{"id":"https://openalex.org/W2411284065","doi":"https://doi.org/10.3727/096368916x690863","title":"Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts","display_name":"Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts","publication_year":2016,"publication_date":"2016-02-05","ids":{"openalex":"https://openalex.org/W2411284065","doi":"https://doi.org/10.3727/096368916x690863","mag":"2411284065","pmid":"https://pubmed.ncbi.nlm.nih.gov/26850319","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/6476541"},"language":"en","primary_location":{"id":"doi:10.3727/096368916x690863","doi":"https://doi.org/10.3727/096368916x690863","is_oa":true,"landing_page_url":"https://doi.org/10.3727/096368916x690863","pdf_url":"https://journals.sagepub.com/doi/pdf/10.3727/096368916X690863","source":{"id":"https://openalex.org/S165333527","display_name":"Cell Transplantation","issn_l":"0963-6897","issn":["0963-6897","1555-3892"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320017","host_organization_name":"SAGE Publishing","host_organization_lineage":["https://openalex.org/P4310320017"],"host_organization_lineage_names":["SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cell Transplantation","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"bronze","oa_url":"https://journals.sagepub.com/doi/pdf/10.3727/096368916X690863","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5015316048","display_name":"Kyle D. Fink","orcid":"https://orcid.org/0000-0002-0235-5038","observed_orcids":["https://orcid.org/0000-0002-0235-5038"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Kyle D. Fink","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5039005607","display_name":"Peter Deng","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Peter Deng","raw_affiliation_strings":["Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, CA, USA","Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, CA, USA","institution_ids":["https://openalex.org/I84218800"]},{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5088262820","display_name":"Joshua Gutierrez","orcid":"https://orcid.org/0009-0008-2595-9484","observed_orcids":["https://orcid.org/0009-0008-2595-9484"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Josh Gutierrez","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5010572953","display_name":"Joseph S. Anderson","orcid":"https://orcid.org/0000-0001-6001-1156","observed_orcids":["https://orcid.org/0000-0001-6001-1156"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joseph S. Anderson","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5079729425","display_name":"Audrey Torrest","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Audrey Torrest","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5053622882","display_name":"Anvita Komarla","orcid":"https://orcid.org/0000-0002-7438-8541","observed_orcids":["https://orcid.org/0000-0002-7438-8541"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Anvita Komarla","raw_affiliation_strings":["Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, CA, USA","Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, CA, USA","institution_ids":["https://openalex.org/I84218800"]},{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5034716695","display_name":"Stefanos Kalomoiris","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Stefanos Kalomoiris","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5016352908","display_name":"Whitney Cary","orcid":"https://orcid.org/0009-0008-4424-6463","observed_orcids":["https://orcid.org/0009-0008-4424-6463"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Whitney Cary","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5072521797","display_name":"Johnathon David Anderson","orcid":"https://orcid.org/0000-0001-5404-2298","observed_orcids":["https://orcid.org/0000-0001-5404-2298"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Johnathon D. Anderson","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5068157325","display_name":"William Gruenloh","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"William Gruenloh","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5082854463","display_name":"Alexandra Duffy","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Alexandra Duffy","raw_affiliation_strings":["Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5009153799","display_name":"Teresa Tempkin","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Teresa Tempkin","raw_affiliation_strings":["Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5108102861","display_name":"Geralyn Annett","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Geralyn Annett","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5064964924","display_name":"Vicki Wheelock","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Vicki Wheelock","raw_affiliation_strings":["Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Neurology, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5089092085","display_name":"David Jay Segal","orcid":"https://orcid.org/0000-0001-8962-3105","observed_orcids":["https://orcid.org/0000-0001-8962-3105"]},"institutions":[{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"David J. Segal","raw_affiliation_strings":["Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Genome Center, MIND Institute, and Biochemistry and Molecular Medicine, University of California, Davis, CA, USA","institution_ids":["https://openalex.org/I84218800"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5085630765","display_name":"Jan A. Nolta","orcid":"https://orcid.org/0000-0003-4576-8542","observed_orcids":["https://orcid.org/0000-0003-4576-8542"]},"institutions":[{"id":"https://openalex.org/I4210160856","display_name":"UC Davis Health","ror":"https://ror.org/05q8kyc69","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210160856"]},{"id":"https://openalex.org/I84218800","display_name":"University of California, Davis","ror":"https://ror.org/05rrcem69","country_code":"US","type":"education","lineage":["https://openalex.org/I84218800"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Jan A. Nolta","raw_affiliation_strings":["Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Stem Cell Program and Institute for Regenerative Cures, University of California Davis Health Systems, Sacramento, CA, USA","institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":2,"corresponding_author_ids":["https://openalex.org/A5015316048"],"corresponding_institution_ids":["https://openalex.org/I4210160856","https://openalex.org/I84218800"],"apc_list":{"value":2650,"currency":"USD","value_usd":2650},"apc_paid":null,"fwci":2.9907,"has_fulltext":true,"cited_by_count":75,"citation_normalized_percentile":{"value":0.90400552,"is_in_top_1_percent":false,"is_in_top_10_percent":true},"cited_by_percentile_year":{"min":94,"max":100},"biblio":{"volume":"25","issue":"4","first_page":"677","last_page":"686"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10949","display_name":"Genetic Neurodegenerative Diseases","score":0.6708999872207642,"subfield":{"id":"https://openalex.org/subfields/2804","display_name":"Cellular and Molecular Neuroscience"},"field":{"id":"https://openalex.org/fields/28","display_name":"Neuroscience"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10949","display_name":"Genetic Neurodegenerative Diseases","score":0.6708999872207642,"subfield":{"id":"https://openalex.org/subfields/2804","display_name":"Cellular and Molecular Neuroscience"},"field":{"id":"https://openalex.org/fields/28","display_name":"Neuroscience"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10878","display_name":"CRISPR and Genetic Engineering","score":0.29739999771118164,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.003000000026077032,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/huntington-s-disease","display_name":"Huntington's disease","score":0.9980000257492065},{"id":"https://openalex.org/keywords/mutant-huntingtin","display_name":"mutant huntingtin","score":0.8450000286102295},{"id":"https://openalex.org/keywords/transcription-activator-like-effectors","display_name":"transcription activator-like effectors","score":0.9800000190734863},{"id":"https://openalex.org/keywords/transcription-activator-like-effector-nucleases","display_name":"TALEN","score":0.8420000076293945},{"id":"https://openalex.org/keywords/cag-repeat-expansion","display_name":"CAG repeat expansion","score":0.7799999713897705},{"id":"https://openalex.org/keywords/polyglutamine","display_name":"polyglutamine","score":0.7250000238418579},{"id":"https://openalex.org/keywords/single-nucleotide-polymorphisms","display_name":"single nucleotide polymorphisms","score":0.8849999904632568},{"id":"https://openalex.org/keywords/fibroblasts","display_name":"fibroblasts","score":0.7110000252723694},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.6039999723434448}],"concepts":[{"id":"https://openalex.org/C2781427258","wikidata":"https://www.wikidata.org/wiki/Q2620886","display_name":"Huntingtin","level":4,"score":0.9085251092910767},{"id":"https://openalex.org/C180754005","wikidata":"https://www.wikidata.org/wiki/Q80726","display_name":"Allele","level":3,"score":0.6688181161880493},{"id":"https://openalex.org/C2780647506","wikidata":"https://www.wikidata.org/wiki/Q190564","display_name":"Huntington's disease","level":3,"score":0.6601343154907227},{"id":"https://openalex.org/C2910011555","wikidata":"https://www.wikidata.org/wiki/Q2620886","display_name":"Huntingtin Protein","level":5,"score":0.5917752981185913},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5382252931594849},{"id":"https://openalex.org/C143065580","wikidata":"https://www.wikidata.org/wiki/Q3285695","display_name":"Mutant","level":3,"score":0.5124819278717041},{"id":"https://openalex.org/C88045685","wikidata":"https://www.wikidata.org/wiki/Q174576","display_name":"Activator (genetics)","level":3,"score":0.5031713843345642},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.4999847412109375},{"id":"https://openalex.org/C51785407","wikidata":"https://www.wikidata.org/wiki/Q2627568","display_name":"Effector","level":2,"score":0.4609440267086029},{"id":"https://openalex.org/C86339819","wikidata":"https://www.wikidata.org/wiki/Q407384","display_name":"Transcription factor","level":3,"score":0.43123072385787964},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3673110604286194},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.3609662652015686},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.2903512716293335},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.22132983803749084},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.16885429620742798},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000071058","descriptor_name":"Huntingtin Protein","qualifier_ui":"Q000096","qualifier_name":"biosynthesis","is_major_topic":true},{"descriptor_ui":"D000071058","descriptor_name":"Huntingtin Protein","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D000483","descriptor_name":"Alleles","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005347","descriptor_name":"Fibroblasts","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006816","descriptor_name":"Huntington Disease","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D006816","descriptor_name":"Huntington Disease","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015533","descriptor_name":"Transcriptional Activation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D020641","descriptor_name":"Polymorphism, Single Nucleotide","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D055785","descriptor_name":"Gene Knockdown Techniques","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":7,"locations":[{"id":"doi:10.3727/096368916x690863","doi":"https://doi.org/10.3727/096368916x690863","is_oa":true,"landing_page_url":"https://doi.org/10.3727/096368916x690863","pdf_url":"https://journals.sagepub.com/doi/pdf/10.3727/096368916X690863","source":{"id":"https://openalex.org/S165333527","display_name":"Cell Transplantation","issn_l":"0963-6897","issn":["0963-6897","1555-3892"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320017","host_organization_name":"SAGE Publishing","host_organization_lineage":["https://openalex.org/P4310320017"],"host_organization_lineage_names":["SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cell Transplantation","raw_type":"journal-article"},{"id":"pmid:26850319","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/26850319","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cell transplantation","raw_type":"Journal Article"},{"id":"pmh:oai:europepmc.org:5458734","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/6476541","pdf_url":null,"source":{"id":"https://openalex.org/S4306400806","display_name":"Europe PMC (PubMed Central)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1303153112","host_organization_name":"European Bioinformatics Institute","host_organization_lineage":["https://openalex.org/I1303153112"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":null,"raw_type":"Text"},{"id":"pmh:oai:pubmedcentral.nih.gov:6476541","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/6476541","pdf_url":null,"source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":"Cell Transplant","raw_type":"Text"},{"id":"pmh:oai:escholarship.org:ark:/13030/qt93x8z46g","doi":null,"is_oa":true,"landing_page_url":"https://escholarship.org/uc/item/93x8z46g","pdf_url":"https://escholarship.org/content/qt93x8z46g/qt93x8z46g.pdf","source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Cell Transplantation, vol 25, iss 4","raw_type":"article"},{"id":"pmh:oai:doaj.org/article:60ffe3d02b6247aa90a1db31550cc403","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/60ffe3d02b6247aa90a1db31550cc403","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Cell Transplantation, Vol 25 (2016)","raw_type":"article"},{"id":"pmh:oai:escholarship.org/ark:/13030/qt93x8z46g","doi":null,"is_oa":false,"landing_page_url":"https://escholarship.org/uc/item/93x8z46g","pdf_url":null,"source":{"id":"https://openalex.org/S4306400115","display_name":"eScholarship (California Digital Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I2801248553","host_organization_name":"California Digital Library","host_organization_lineage":["https://openalex.org/I2801248553"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Cell transplantation, vol 25, iss 4","raw_type":"article"}],"best_oa_location":{"id":"doi:10.3727/096368916x690863","doi":"https://doi.org/10.3727/096368916x690863","is_oa":true,"landing_page_url":"https://doi.org/10.3727/096368916x690863","pdf_url":"https://journals.sagepub.com/doi/pdf/10.3727/096368916X690863","source":{"id":"https://openalex.org/S165333527","display_name":"Cell Transplantation","issn_l":"0963-6897","issn":["0963-6897","1555-3892"],"is_oa":false,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-1","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320017","host_organization_name":"SAGE Publishing","host_organization_lineage":["https://openalex.org/P4310320017"],"host_organization_lineage_names":["SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cell Transplantation","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","score":0.9391000270843506,"id":"https://metadata.un.org/sdg/3"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G1128517185","display_name":"Training Program in Biomolecular Technology","funder_award_id":"T32GM008799","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G1368571601","display_name":"Novel epigenetic targets for silencing the Huntingtons disease mutation","funder_award_id":"F32NS090722","funder_id":"https://openalex.org/F4320337359","funder_display_name":"National Institute of Neurological Disorders and Stroke"},{"id":"https://openalex.org/G1751591096","display_name":"Predoctoral Training in Pharmacological Sciences","funder_award_id":"T32GM099608","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G2033686746","display_name":null,"funder_award_id":"RR 026825","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G2137968735","display_name":null,"funder_award_id":"NCRR C06-RR12088","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G2184793071","display_name":null,"funder_award_id":"C06-RR012088","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G327948365","display_name":null,"funder_award_id":"C06 RR 12088","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G3641269095","display_name":"Direct cell to cell transfer of microRNA for tissue repair","funder_award_id":"R01GM099688","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G4043104618","display_name":null,"funder_award_id":"RR12964","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G5778689702","display_name":null,"funder_award_id":"CA0933730","funder_id":"https://openalex.org/F4320337351","funder_display_name":"National Cancer Institute"},{"id":"https://openalex.org/G6215400813","display_name":"EXTRAMURAL RESEARCH FACILITIES CONSTRUCTION","funder_award_id":"1c06rr012088-01","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G7997815769","display_name":null,"funder_award_id":"S10 RR12964","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G8012802046","display_name":"NextGen DNA Binding Proteins Based on TAL Domains","funder_award_id":"R01GM097073","funder_id":"https://openalex.org/F4320337354","funder_display_name":"National Institute of General Medical Sciences"},{"id":"https://openalex.org/G8132083788","display_name":"CELL SORTER FOR AN OPTICAL BIOLOGY FACILITY","funder_award_id":"1s10rr012964-01","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G8157273241","display_name":"Shared LSRII Cytometer Equipment Application - UC Davis Stem Cell Program","funder_award_id":"S10RR026825","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G8271676383","display_name":"Novel immune deficient mouse models of Huntington's disease","funder_award_id":"R24OD021606","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G8501212149","display_name":null,"funder_award_id":"RR12088","funder_id":"https://openalex.org/F4320337375","funder_display_name":"National Center for Research Resources"},{"id":"https://openalex.org/G9000858444","display_name":"Training Program in Basic and Translational Cardiovascular Science","funder_award_id":"T32HL086350","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G9000917502","display_name":"MSCS ENGINEERED TO PRODUCE BDNF AND GENE EDITING CARGO FOR THE TREATMENT OF HUNTINGTON'S DISEASE","funder_award_id":"R01NS102486","funder_id":"https://openalex.org/F4320337359","funder_display_name":"National Institute of Neurological Disorders and Stroke"},{"id":"https://openalex.org/G952886752","display_name":"MSC engineered to produce BDNF for the treatment of Huntington\u2019s disease","funder_award_id":"DR2-05415","funder_id":"https://openalex.org/F4320306171","funder_display_name":"California Institute for Regenerative Medicine"}],"funders":[{"id":"https://openalex.org/F4320306076","display_name":"National Science Foundation","ror":"https://ror.org/021nxhr62"},{"id":"https://openalex.org/F4320306171","display_name":"California Institute for Regenerative Medicine","ror":"https://ror.org/033m8b439"},{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"},{"id":"https://openalex.org/F4320337338","display_name":"National Heart, Lung, and Blood Institute","ror":"https://ror.org/012pb6c26"},{"id":"https://openalex.org/F4320337351","display_name":"National Cancer Institute","ror":"https://ror.org/040gcmg81"},{"id":"https://openalex.org/F4320337354","display_name":"National Institute of General Medical Sciences","ror":"https://ror.org/04q48ey07"},{"id":"https://openalex.org/F4320337359","display_name":"National Institute of Neurological Disorders and Stroke","ror":"https://ror.org/01s5ya894"},{"id":"https://openalex.org/F4320337375","display_name":"National Center for Research Resources","ror":"https://ror.org/04pw6fb54"}],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W2411284065.pdf","grobid_xml":"https://content.openalex.org/works/W2411284065.grobid-xml"},"referenced_works_count":51,"referenced_works":["https://openalex.org/W39675397","https://openalex.org/W1543664114","https://openalex.org/W1547102031","https://openalex.org/W1809261872","https://openalex.org/W1836009684","https://openalex.org/W1895398290","https://openalex.org/W1911766697","https://openalex.org/W1963840240","https://openalex.org/W1967951176","https://openalex.org/W1981872108","https://openalex.org/W1990319434","https://openalex.org/W1992305831","https://openalex.org/W1997188614","https://openalex.org/W1997369314","https://openalex.org/W2008784634","https://openalex.org/W2011035933","https://openalex.org/W2019480042","https://openalex.org/W2020117714","https://openalex.org/W2024436194","https://openalex.org/W2027385910","https://openalex.org/W2036038695","https://openalex.org/W2045905604","https://openalex.org/W2046855179","https://openalex.org/W2047121554","https://openalex.org/W2047148496","https://openalex.org/W2048000239","https://openalex.org/W2053778058","https://openalex.org/W2056793372","https://openalex.org/W2057758054","https://openalex.org/W2062364641","https://openalex.org/W2062521097","https://openalex.org/W2064886170","https://openalex.org/W2069132567","https://openalex.org/W2069686570","https://openalex.org/W2077225885","https://openalex.org/W2082579696","https://openalex.org/W2083045704","https://openalex.org/W2085618939","https://openalex.org/W2098123284","https://openalex.org/W2098248120","https://openalex.org/W2099040545","https://openalex.org/W2112006537","https://openalex.org/W2121642311","https://openalex.org/W2132082584","https://openalex.org/W2152102410","https://openalex.org/W2161311076","https://openalex.org/W2164675092","https://openalex.org/W2168245648","https://openalex.org/W2321362142","https://openalex.org/W2404071039","https://openalex.org/W4230475322"],"related_works":["https://openalex.org/W2048030733","https://openalex.org/W2124654811","https://openalex.org/W2953448192","https://openalex.org/W1998080723","https://openalex.org/W3045392103","https://openalex.org/W4403362914","https://openalex.org/W2310346606","https://openalex.org/W4249413707","https://openalex.org/W2029900342","https://openalex.org/W7097087544"],"abstract_inverted_index":{"Huntington's":[0,207],"disease":[1],"(HD)":[2],"is":[3],"an":[4,11,61],"autosomal":[5],"dominant":[6],"neurodegenerative":[7],"disorder":[8],"caused":[9],"by":[10],"abnormal":[12],"expansion":[13],"of":[14,47,99,180,189],"CAG":[15],"repeats.":[16],"Although":[17],"pathogenesis":[18],"has":[19,41],"been":[20,42],"attributed":[21],"to":[22,37,59,78,95,117,123,173],"this":[23],"polyglutamine":[24],"expansion,":[25],"the":[26,31,84,100,128,181,187],"underlying":[27],"mechanisms":[28],"through":[29,50],"which":[30],"huntingtin":[32,49],"protein":[33,51,150],"functions":[34],"have":[35],"yet":[36],"be":[38,60,118],"elucidated.":[39],"It":[40],"suggested":[43],"that":[44],"postnatal":[45],"reduction":[46],"mutant":[48,85,129,149,165],"interference":[52],"or":[53,139,208],"conditional":[54],"gene":[55,191],"knockout":[56],"could":[57],"prove":[58],"effective":[62],"therapy":[63],"for":[64,157,200,203],"patients":[65],"suffering":[66,205],"from":[67,206],"HD.":[68],"For":[69],"allele-specific":[70,190],"targeting,":[71],"transcription":[72],"activator-like":[73],"effectors":[74],"(TALE)":[75],"were":[76,105,115,134],"designed":[77,116],"target":[79],"single-nucleotide":[80],"polymorphisms":[81],"(SNP)":[82],"in":[83,127],"allele":[86,166],"and":[87,114,148,161,196],"packaged":[88,106],"into":[89,107],"a":[90,108,125,145,198],"vector":[91,109],"backbone":[92,110],"containing":[93,111],"KRAB":[94],"promote":[96],"transcriptional":[97],"repression":[98],"disease-associated":[101],"allele.":[102,130,183],"Additional":[103],"TALEs":[104],"heterodimeric":[112],"FokI":[113],"used":[119],"as":[120],"nucleases":[121],"(TALEN)":[122],"cause":[124],"CAG-collapse":[126],"Human":[131],"HD":[132],"fibroblasts":[133],"treated":[135],"with":[136,154],"each":[137],"TALE-SNP":[138,160],"TALEN.":[140],"Allele-expression":[141],"was":[142,152],"measured":[143],"using":[144,193],"SNP-genotyping":[146],"assay":[147],"aggregation":[151],"quantified":[153],"Western":[155],"blots":[156],"anti-ubiquitin.":[158],"The":[159],"TALEN":[162],"significantly":[163],"reduced":[164],"expression":[167,179],"(p":[168],"<":[169],"0.05)":[170],"when":[171],"compared":[172],"control":[174],"transfections":[175],"while":[176],"not":[177],"affecting":[178],"nondisease":[182],"This":[184],"study":[185],"demonstrates":[186],"potential":[188],"modification":[192],"TALE":[194],"proteins,":[195],"provides":[197],"foundation":[199],"targeted":[201],"treatment":[202],"individuals":[204],"other":[209],"genetically":[210],"linked":[211],"diseases.":[212]},"counts_by_year":[{"year":2026,"cited_by_count":3},{"year":2025,"cited_by_count":7},{"year":2024,"cited_by_count":16},{"year":2023,"cited_by_count":6},{"year":2022,"cited_by_count":11},{"year":2021,"cited_by_count":9},{"year":2020,"cited_by_count":7},{"year":2019,"cited_by_count":10},{"year":2018,"cited_by_count":2},{"year":2017,"cited_by_count":4}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2016-06-24T00:00:00"}