{"id":"https://openalex.org/W2031328131","doi":"https://doi.org/10.1073/pnas.0912896107","title":"Design of embedded chimeric peptide nucleic acids that efficiently enter and accurately reactivate gene expression in vivo","display_name":"Design of embedded chimeric peptide nucleic acids that efficiently enter and accurately reactivate gene expression in vivo","publication_year":2010,"publication_date":"2010-09-13","ids":{"openalex":"https://openalex.org/W2031328131","doi":"https://doi.org/10.1073/pnas.0912896107","mag":"2031328131","pmid":"https://pubmed.ncbi.nlm.nih.gov/20837550","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/2947906"},"language":"en","primary_location":{"id":"doi:10.1073/pnas.0912896107","doi":"https://doi.org/10.1073/pnas.0912896107","is_oa":false,"landing_page_url":"https://doi.org/10.1073/pnas.0912896107","pdf_url":null,"source":{"id":"https://openalex.org/S125754415","display_name":"Proceedings of the National Academy of Sciences","issn_l":"0027-8424","issn":["0027-8424","1091-6490"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","erih-plus","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320052","host_organization_name":"National Academy of Sciences","host_organization_lineage":["https://openalex.org/P4310320052"],"host_organization_lineage_names":["National Academy of Sciences"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Proceedings of the National Academy of Sciences","raw_type":"journal-article"},"volume":null,"type":"article","indexed_in":["crossref","pubmed"],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/2947906","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5084674068","display_name":"Joy Chen","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I98704320","display_name":"Icahn School of Medicine at Mount Sinai","ror":"https://ror.org/04a9tmd77","country_code":"US","type":"education","lineage":["https://openalex.org/I1320796813","https://openalex.org/I98704320"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Joy Chen","raw_affiliation_strings":["Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY, 10029;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY, 10029;","institution_ids":["https://openalex.org/I98704320"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5001750325","display_name":"Kenneth R. Peterson","orcid":"https://orcid.org/0000-0002-8598-9334","observed_orcids":["https://orcid.org/0000-0002-8598-9334"]},"institutions":[{"id":"https://openalex.org/I4210128618","display_name":"University of Kansas Medical Center","ror":"https://ror.org/036c9yv20","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I4210128618"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kenneth R. Peterson","raw_affiliation_strings":["Department of Biochemistry and Molecular Biology, University of Kansas Medical Center, Kansas City, KS 66160;"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Biochemistry and Molecular Biology, University of Kansas Medical Center, Kansas City, KS 66160;","institution_ids":["https://openalex.org/I4210128618"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5026108947","display_name":"Camelia Iancu\u2010Rubin","orcid":"https://orcid.org/0000-0002-3796-3332","observed_orcids":["https://orcid.org/0000-0002-3796-3332"]},"institutions":[{"id":"https://openalex.org/I4390039328","display_name":"Tisch Cancer Institute","ror":"https://ror.org/0317dzj93","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1320796813","https://openalex.org/I4390039328","https://openalex.org/I98704320"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Camelia Iancu-Rubin","raw_affiliation_strings":["Department of Medicine,","Tisch Cancer Institute, and","Department of Medicine,; Tisch Cancer Institute, and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Medicine,","institution_ids":[]},{"raw_affiliation_string":"Tisch Cancer Institute, and","institution_ids":["https://openalex.org/I4390039328"]},{"raw_affiliation_string":"Department of Medicine,; Tisch Cancer Institute, and","institution_ids":["https://openalex.org/I4390039328"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5002966230","display_name":"James J. Bieker","orcid":"https://orcid.org/0000-0001-5128-7476","observed_orcids":["https://orcid.org/0000-0001-5128-7476"]},"institutions":[{"id":"https://openalex.org/I4390039328","display_name":"Tisch Cancer Institute","ror":"https://ror.org/0317dzj93","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1320796813","https://openalex.org/I4390039328","https://openalex.org/I98704320"]},{"id":"https://openalex.org/I98704320","display_name":"Icahn School of Medicine at Mount Sinai","ror":"https://ror.org/04a9tmd77","country_code":"US","type":"education","lineage":["https://openalex.org/I1320796813","https://openalex.org/I98704320"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"James J. Bieker","raw_affiliation_strings":["Black Family Stem Cell Institute, Mount Sinai School of Medicine, New York, NY, 10029","Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY, 10029;","Tisch Cancer Institute, and","Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY, 10029;; Tisch Cancer Institute, and"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Black Family Stem Cell Institute, Mount Sinai School of Medicine, New York, NY, 10029","institution_ids":["https://openalex.org/I98704320"]},{"raw_affiliation_string":"Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY, 10029;","institution_ids":["https://openalex.org/I98704320"]},{"raw_affiliation_string":"Tisch Cancer Institute, and","institution_ids":["https://openalex.org/I4390039328"]},{"raw_affiliation_string":"Department of Developmental and Regenerative Biology, Mount Sinai School of Medicine, New York, NY, 10029;; Tisch Cancer Institute, and","institution_ids":["https://openalex.org/I4390039328","https://openalex.org/I98704320"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":0.6519,"has_fulltext":false,"cited_by_count":16,"citation_normalized_percentile":{"value":0.65590498,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":97},"biblio":{"volume":"107","issue":"39","first_page":"16846","last_page":"16851"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.9467999935150146,"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"}},"topics":[{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":0.9467999935150146,"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/T10432","display_name":"DNA and Nucleic Acid Chemistry","score":0.021199999377131462,"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/T10554","display_name":"Hemoglobinopathies and Related Disorders","score":0.007600000128149986,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/peptide-nucleic-acid","display_name":"peptide nucleic acid","score":0.8769999742507935},{"id":"https://openalex.org/keywords/transcriptional-reactivation","display_name":"transcriptional reactivation","score":0.9020000100135803},{"id":"https://openalex.org/keywords/hemoglobinopathies","display_name":"hemoglobinopathies","score":0.8539999723434448},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.48899999260902405},{"id":"https://openalex.org/keywords/nuclear-entry","display_name":"nuclear entry","score":0.5260000228881836},{"id":"https://openalex.org/keywords/cell-penetrating-peptides","display_name":"cell-penetrating peptides","score":0.6069999933242798},{"id":"https://openalex.org/keywords/dna-binding","display_name":"DNA binding","score":0.5120000243186951},{"id":"https://openalex.org/keywords/live-cell-imaging","display_name":"live-cell imaging","score":0.7770000100135803},{"id":"https://openalex.org/keywords/transcriptional-activator","display_name":"transcriptional activator","score":0.5230000019073486}],"concepts":[{"id":"https://openalex.org/C24107716","wikidata":"https://www.wikidata.org/wiki/Q123619","display_name":"Nucleic acid","level":2,"score":0.6155833005905151},{"id":"https://openalex.org/C207001950","wikidata":"https://www.wikidata.org/wiki/Q141124","display_name":"In vivo","level":2,"score":0.605709969997406},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.5190116167068481},{"id":"https://openalex.org/C202751555","wikidata":"https://www.wikidata.org/wiki/Q221681","display_name":"In vitro","level":2,"score":0.4609750807285309},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.45983263850212097},{"id":"https://openalex.org/C552990157","wikidata":"https://www.wikidata.org/wiki/Q7430","display_name":"DNA","level":2,"score":0.45139971375465393},{"id":"https://openalex.org/C2779281246","wikidata":"https://www.wikidata.org/wiki/Q172847","display_name":"Peptide","level":2,"score":0.43395522236824036},{"id":"https://openalex.org/C150194340","wikidata":"https://www.wikidata.org/wiki/Q26972","display_name":"Gene expression","level":3,"score":0.4261898398399353},{"id":"https://openalex.org/C102230213","wikidata":"https://www.wikidata.org/wiki/Q430117","display_name":"Transgene","level":3,"score":0.42050570249557495},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.4152616858482361},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.3437856435775757},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.331508070230484},{"id":"https://openalex.org/C70721500","wikidata":"https://www.wikidata.org/wiki/Q177005","display_name":"Computational biology","level":1,"score":0.33006006479263306},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.20321327447891235}],"mesh":[{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D001854","descriptor_name":"Bone Marrow Cells","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D001854","descriptor_name":"Bone Marrow Cells","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D001854","descriptor_name":"Bone Marrow Cells","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D002460","descriptor_name":"Cell Line","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002460","descriptor_name":"Cell Line","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002460","descriptor_name":"Cell Line","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002467","descriptor_name":"Cell Nucleus","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D002467","descriptor_name":"Cell Nucleus","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D002467","descriptor_name":"Cell Nucleus","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D002478","descriptor_name":"Cells, Cultured","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002478","descriptor_name":"Cells, Cultured","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D002478","descriptor_name":"Cells, Cultured","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006453","descriptor_name":"Hemoglobinopathies","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":false},{"descriptor_ui":"D006453","descriptor_name":"Hemoglobinopathies","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":false},{"descriptor_ui":"D006453","descriptor_name":"Hemoglobinopathies","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":false},{"descriptor_ui":"D006453","descriptor_name":"Hemoglobinopathies","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D006453","descriptor_name":"Hemoglobinopathies","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D006453","descriptor_name":"Hemoglobinopathies","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D011401","descriptor_name":"Promoter Regions, Genetic","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D011401","descriptor_name":"Promoter Regions, Genetic","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D011401","descriptor_name":"Promoter Regions, Genetic","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D015195","descriptor_name":"Drug Design","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D015195","descriptor_name":"Drug Design","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D015195","descriptor_name":"Drug Design","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D015870","descriptor_name":"Gene Expression","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D015870","descriptor_name":"Gene Expression","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D015870","descriptor_name":"Gene Expression","qualifier_ui":"Q000187","qualifier_name":"drug effects","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000494","qualifier_name":"pharmacology","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000737","qualifier_name":"chemistry","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000737","qualifier_name":"chemistry","is_major_topic":false},{"descriptor_ui":"D020135","descriptor_name":"Peptide Nucleic Acids","qualifier_ui":"Q000737","qualifier_name":"chemistry","is_major_topic":false},{"descriptor_ui":"D047369","descriptor_name":"Chromatin Immunoprecipitation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D047369","descriptor_name":"Chromatin Immunoprecipitation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D047369","descriptor_name":"Chromatin Immunoprecipitation","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D051379","descriptor_name":"Mice","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D055545","descriptor_name":"gamma-Globins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D055545","descriptor_name":"gamma-Globins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D055545","descriptor_name":"gamma-Globins","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1073/pnas.0912896107","doi":"https://doi.org/10.1073/pnas.0912896107","is_oa":false,"landing_page_url":"https://doi.org/10.1073/pnas.0912896107","pdf_url":null,"source":{"id":"https://openalex.org/S125754415","display_name":"Proceedings of the National Academy of Sciences","issn_l":"0027-8424","issn":["0027-8424","1091-6490"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-class-a","anvur-scientific","cwts-core","dongbi-a","doyens","erih-plus","jufo-3","ki-jl-3","medline","norway-2","poland-200","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320052","host_organization_name":"National Academy of Sciences","host_organization_lineage":["https://openalex.org/P4310320052"],"host_organization_lineage_names":["National Academy of Sciences"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Proceedings of the National Academy of Sciences","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:2947906","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/2947906","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":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Proc Natl Acad Sci U S A","raw_type":"Text"},{"id":"pmid:20837550","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/20837550","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":"Proceedings of the National Academy of Sciences of the United States of America","raw_type":"Journal Article"}],"best_oa_location":{"id":"pmh:oai:pubmedcentral.nih.gov:2947906","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/2947906","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":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Proc Natl Acad Sci U S A","raw_type":"Text"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.6240000128746033}],"sustainable_development_goals_aurora":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.6200000047683716}],"study_designs":[],"awards":[{"id":"https://openalex.org/G4451553129","display_name":"PNA-based strategies to reverse gamma-globin gene silencing*","funder_award_id":"R01HL073437","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G595883429","display_name":"Acquisition of Confocal Laser Scanning Microscope for       Institutional Core Imaging Facility","funder_award_id":"9724504","funder_id":"https://openalex.org/F4320306076","funder_display_name":"National Science Foundation"},{"id":"https://openalex.org/G6149236568","display_name":"Advanced Microscopy Shared Resource Facility","funder_award_id":"R24CA095823","funder_id":"https://openalex.org/F4320337351","funder_display_name":"National Cancer Institute"},{"id":"https://openalex.org/G644865321","display_name":null,"funder_award_id":"AP20187","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G7500932798","display_name":null,"funder_award_id":"HL73437","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"}],"funders":[{"id":"https://openalex.org/F4320306076","display_name":"National Science Foundation","ror":"https://ror.org/021nxhr62"},{"id":"https://openalex.org/F4320309021","display_name":"Ariad Pharmaceuticals","ror":"https://ror.org/01dwtyk53"},{"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"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":41,"referenced_works":["https://openalex.org/W132410613","https://openalex.org/W1521801418","https://openalex.org/W1531811400","https://openalex.org/W1579482787","https://openalex.org/W1861971834","https://openalex.org/W1975500382","https://openalex.org/W1977484661","https://openalex.org/W1977696694","https://openalex.org/W1981464117","https://openalex.org/W1988286575","https://openalex.org/W1989018215","https://openalex.org/W2005752061","https://openalex.org/W2007578481","https://openalex.org/W2016788088","https://openalex.org/W2019818589","https://openalex.org/W2021210659","https://openalex.org/W2024537855","https://openalex.org/W2037598951","https://openalex.org/W2044511711","https://openalex.org/W2048249977","https://openalex.org/W2048330855","https://openalex.org/W2051745799","https://openalex.org/W2052426861","https://openalex.org/W2057536036","https://openalex.org/W2061867662","https://openalex.org/W2063853405","https://openalex.org/W2066804477","https://openalex.org/W2086614811","https://openalex.org/W2090471601","https://openalex.org/W2101310061","https://openalex.org/W2103262726","https://openalex.org/W2108041597","https://openalex.org/W2120959775","https://openalex.org/W2130389986","https://openalex.org/W2151682372","https://openalex.org/W2160863389","https://openalex.org/W2166828381","https://openalex.org/W2334263927","https://openalex.org/W2399658643","https://openalex.org/W3158286353","https://openalex.org/W4246892345"],"related_works":["https://openalex.org/W1968079539","https://openalex.org/W3025337362","https://openalex.org/W2016788088","https://openalex.org/W10373600","https://openalex.org/W2010884996","https://openalex.org/W2140166099","https://openalex.org/W7155160555","https://openalex.org/W2039806483","https://openalex.org/W1971054272","https://openalex.org/W7204949406"],"abstract_inverted_index":{"Pharmacological":[0],"treatments":[1,28],"designed":[2],"to":[3,9,46,64,105,115,136,159,172,183],"reactivate":[4],"fetal":[5,71],"\u03b3-globin":[6,72],"can":[7,169],"lead":[8,114],"an":[10,48],"effective":[11,32],"and":[12,36,60,74,84,92,107,140,155,178],"successful":[13,116],"clinical":[14,184],"outcome":[15],"in":[16,78,93,97,163],"patients":[17],"with":[18,99],"hemoglobinopathies.":[19],"However,":[20],"new":[21],"approaches":[22],"remain":[23],"highly":[24],"desired":[25],"because":[26],"such":[27,190],"are":[29,156,179],"not":[30],"equally":[31],"for":[33],"all":[34],"patients,":[35],"toxicity":[37],"issues":[38],"remain.":[39],"We":[40],"have":[41,102],"taken":[42],"a":[43,123,191],"systematic":[44],"approach":[45],"develop":[47],"embedded":[49,128,150],"chimeric":[50,109],"peptide":[51],"nucleic":[52],"acid":[53,132],"(PNA)":[54],"that":[55,113,168,186],"effectively":[56],"enters":[57],"the":[58,61,69,141,147,160],"cell":[59],"nucleus,":[62],"binds":[63],"its":[65],"target":[66],"site":[67],"at":[68],"human":[70,85],"promoter,":[73],"reactivates":[75],"this":[76],"transcript":[77],"adult":[79],"transgenic":[80],"mouse":[81],"bone":[82],"marrow":[83],"primary":[86],"peripheral":[87],"blood":[88],"cells.":[89],"In":[90],"vitro":[91],"vivo":[94,164],"DNA-binding":[95],"assays":[96],"conjunction":[98],"live-cell":[100],"imaging":[101],"been":[103],"used":[104],"establish":[106],"optimize":[108],"PNA":[110,126],"design":[111,161],"parameters":[112],"gene":[117],"activation.":[118],"Our":[119],"final":[120],"molecule":[121],"contains":[122],"specific":[124],"\u03b3-promoter-binding":[125],"sequence":[127],"within":[129],"two":[130],"amino":[131],"motifs:":[133],"one":[134],"leads":[135],"efficient":[137],"cell/nuclear":[138],"entry,":[139],"other":[142],"generates":[143],"transcriptional":[144,165],"reactivation":[145],"of":[146,162,176,180],"target.":[148],"These":[149],"PNAs":[151],"overcome":[152],"previous":[153],"limitations":[154],"generally":[157],"applicable":[158],"activation":[166],"reagents":[167],"be":[170],"directed":[171],"any":[173],"promoter":[174],"region":[175],"interest":[177],"direct":[181],"relevance":[182],"applications":[185],"would":[187],"benefit":[188],"from":[189],"need.":[192]},"counts_by_year":[{"year":2022,"cited_by_count":1},{"year":2021,"cited_by_count":2},{"year":2020,"cited_by_count":3},{"year":2019,"cited_by_count":2},{"year":2018,"cited_by_count":1},{"year":2017,"cited_by_count":1},{"year":2016,"cited_by_count":1},{"year":2013,"cited_by_count":1},{"year":2012,"cited_by_count":3}],"updated_date":"2026-10-30T12:39:25.321706","created_date":"2016-06-24T00:00:00"}