{"id":"https://openalex.org/W3014371970","doi":"https://doi.org/10.1089/hum.2020.049","title":"Adeno-Associated Virus and Hematopoietic Stem Cells: The Potential of Adeno-Associated Virus Hematopoietic Stem Cells in Genetic Medicines","display_name":"Adeno-Associated Virus and Hematopoietic Stem Cells: The Potential of Adeno-Associated Virus Hematopoietic Stem Cells in Genetic Medicines","publication_year":2020,"publication_date":"2020-04-07","ids":{"openalex":"https://openalex.org/W3014371970","doi":"https://doi.org/10.1089/hum.2020.049","mag":"3014371970","pmid":"https://pubmed.ncbi.nlm.nih.gov/32253938","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/7232699"},"language":"en","primary_location":{"id":"doi:10.1089/hum.2020.049","doi":"https://doi.org/10.1089/hum.2020.049","is_oa":false,"landing_page_url":"https://doi.org/10.1089/hum.2020.049","pdf_url":null,"source":{"id":"https://openalex.org/S39730559","display_name":"Human Gene Therapy","issn_l":"1043-0342","issn":["1043-0342","1557-7422"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320443","host_organization_name":"Mary Ann Liebert, Inc.","host_organization_lineage":["https://openalex.org/P4310320443","https://openalex.org/P4310320017"],"host_organization_lineage_names":["Mary Ann Liebert, Inc.","SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Human Gene Therapy","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/7232699","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5112802350","display_name":"Saswati Chatterjee","orcid":"https://orcid.org/0009-0004-8568-1227","observed_orcids":["https://orcid.org/0009-0004-8568-1227"]},"institutions":[{"id":"https://openalex.org/I1301076528","display_name":"City Of Hope National Medical Center","ror":"https://ror.org/00w6g5w60","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1301076528"]},{"id":"https://openalex.org/I4210110459","display_name":"City of Hope","ror":"https://ror.org/01z1vct10","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1301076528","https://openalex.org/I4210110459"]},{"id":"https://openalex.org/I4387156432","display_name":"Beckman Research Institute","ror":"https://ror.org/05fazth07","country_code":"US","type":"facility","lineage":["https://openalex.org/I1301076528","https://openalex.org/I4387156432"]}],"countries":["US"],"is_corresponding":true,"raw_author_name":"Saswati Chatterjee","raw_affiliation_strings":["Department of Surgery, Beckman Research Institute of City of Hope Medical Center, Duarte, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Surgery, Beckman Research Institute of City of Hope Medical Center, Duarte, California, USA","institution_ids":["https://openalex.org/I1301076528","https://openalex.org/I4210110459","https://openalex.org/I4387156432"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5080140459","display_name":"Venkatesh Sivanandam","orcid":"https://orcid.org/0000-0001-6017-350X","observed_orcids":["https://orcid.org/0000-0001-6017-350X"]},"institutions":[{"id":"https://openalex.org/I1301076528","display_name":"City Of Hope National Medical Center","ror":"https://ror.org/00w6g5w60","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1301076528"]},{"id":"https://openalex.org/I4210110459","display_name":"City of Hope","ror":"https://ror.org/01z1vct10","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1301076528","https://openalex.org/I4210110459"]},{"id":"https://openalex.org/I4387156432","display_name":"Beckman Research Institute","ror":"https://ror.org/05fazth07","country_code":"US","type":"facility","lineage":["https://openalex.org/I1301076528","https://openalex.org/I4387156432"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Venkatesh Sivanandam","raw_affiliation_strings":["Department of Surgery, Beckman Research Institute of City of Hope Medical Center, Duarte, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Surgery, Beckman Research Institute of City of Hope Medical Center, Duarte, California, USA","institution_ids":["https://openalex.org/I1301076528","https://openalex.org/I4210110459","https://openalex.org/I4387156432"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5108455786","display_name":"Kamehameha K. Wong","orcid":null,"observed_orcids":[]},"institutions":[{"id":"https://openalex.org/I1301076528","display_name":"City Of Hope National Medical Center","ror":"https://ror.org/00w6g5w60","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1301076528"]},{"id":"https://openalex.org/I4210110459","display_name":"City of Hope","ror":"https://ror.org/01z1vct10","country_code":"US","type":"healthcare","lineage":["https://openalex.org/I1301076528","https://openalex.org/I4210110459"]}],"countries":["US"],"is_corresponding":false,"raw_author_name":"Kamehameha Kai-Min Wong","raw_affiliation_strings":["Department of Hematology and Stem Cell Transplantation, City of Hope Medical Center, Duarte, California, USA"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Department of Hematology and Stem Cell Transplantation, City of Hope Medical Center, Duarte, California, USA","institution_ids":["https://openalex.org/I1301076528","https://openalex.org/I4210110459"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":3,"corresponding_author_ids":["https://openalex.org/A5112802350"],"corresponding_institution_ids":["https://openalex.org/I1301076528","https://openalex.org/I4210110459","https://openalex.org/I4387156432"],"apc_list":null,"apc_paid":null,"fwci":0.7323,"has_fulltext":false,"cited_by_count":16,"citation_normalized_percentile":{"value":0.66798222,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":89,"max":99},"biblio":{"volume":"31","issue":"9-10","first_page":"542","last_page":"552"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9994999766349792,"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"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.9994999766349792,"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"}},{"id":"https://openalex.org/T10725","display_name":"RNA Interference and Gene Delivery","score":9.999999747378752e-05,"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/T11280","display_name":"Hemophilia Treatment and Research","score":0.0,"subfield":{"id":"https://openalex.org/subfields/2720","display_name":"Hematology"},"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/adeno-associated-virus","display_name":"adeno-associated virus","score":0.9580000042915344},{"id":"https://openalex.org/keywords/hematopoietic-stem-cells","display_name":"hematopoietic stem cells","score":0.9359999895095825},{"id":"https://openalex.org/keywords/aav2-vector","display_name":"AAV2 vector","score":0.8420000076293945},{"id":"https://openalex.org/keywords/gene-therapy-vectors","display_name":"gene therapy vectors","score":0.6840000152587891},{"id":"https://openalex.org/keywords/homologous-recombination","display_name":"homologous recombination","score":0.8349999785423279},{"id":"https://openalex.org/keywords/phenylketonuria","display_name":"phenylketonuria","score":0.7950000166893005},{"id":"https://openalex.org/keywords/genetic-disorders","display_name":"genetic disorders","score":0.4129999876022339},{"id":"https://openalex.org/keywords/genome-editing","display_name":"genome editing","score":0.6370000243186951},{"id":"https://openalex.org/keywords/blood-brain-barrier-penetration","display_name":"blood-brain barrier penetration","score":0.6129999756813049},{"id":"https://openalex.org/keywords/cd34-cells","display_name":"CD34+ cells","score":0.5960000157356262}],"concepts":[{"id":"https://openalex.org/C2778107364","wikidata":"https://www.wikidata.org/wiki/Q355989","display_name":"Adeno-associated virus","level":5,"score":0.8125668168067932},{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.7393472194671631},{"id":"https://openalex.org/C28328180","wikidata":"https://www.wikidata.org/wiki/Q48196","display_name":"Stem cell","level":2,"score":0.722034752368927},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.7101701498031616},{"id":"https://openalex.org/C109159458","wikidata":"https://www.wikidata.org/wiki/Q919283","display_name":"Haematopoiesis","level":3,"score":0.6248362064361572},{"id":"https://openalex.org/C15152581","wikidata":"https://www.wikidata.org/wiki/Q7833966","display_name":"Transduction (biophysics)","level":2,"score":0.6064916253089905},{"id":"https://openalex.org/C2778086970","wikidata":"https://www.wikidata.org/wiki/Q514525","display_name":"Hematopoietic stem cell","level":4,"score":0.48387253284454346},{"id":"https://openalex.org/C32470452","wikidata":"https://www.wikidata.org/wiki/Q2298489","display_name":"Viral vector","level":4,"score":0.44368916749954224},{"id":"https://openalex.org/C33602714","wikidata":"https://www.wikidata.org/wiki/Q7809666","display_name":"Tissue tropism","level":4,"score":0.44356322288513184},{"id":"https://openalex.org/C92087593","wikidata":"https://www.wikidata.org/wiki/Q427389","display_name":"Vector (molecular biology)","level":4,"score":0.42301011085510254},{"id":"https://openalex.org/C10205521","wikidata":"https://www.wikidata.org/wiki/Q4035578","display_name":"CD34","level":3,"score":0.4208817481994629},{"id":"https://openalex.org/C35693153","wikidata":"https://www.wikidata.org/wiki/Q239762","display_name":"Tropism","level":3,"score":0.4183202385902405},{"id":"https://openalex.org/C144501496","wikidata":"https://www.wikidata.org/wiki/Q5533489","display_name":"Genome editing","level":4,"score":0.4161822199821472},{"id":"https://openalex.org/C2779389132","wikidata":"https://www.wikidata.org/wiki/Q1533033","display_name":"Stem-cell therapy","level":3,"score":0.4113752841949463},{"id":"https://openalex.org/C2522874641","wikidata":"https://www.wikidata.org/wiki/Q808","display_name":"Virus","level":2,"score":0.3252718448638916},{"id":"https://openalex.org/C159047783","wikidata":"https://www.wikidata.org/wiki/Q7215","display_name":"Virology","level":1,"score":0.28037482500076294},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.2545431852340698},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.21664583683013916},{"id":"https://openalex.org/C141231307","wikidata":"https://www.wikidata.org/wiki/Q7020","display_name":"Genome","level":3,"score":0.20704007148742676},{"id":"https://openalex.org/C40767141","wikidata":"https://www.wikidata.org/wiki/Q285697","display_name":"Recombinant DNA","level":3,"score":0.0},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.0}],"mesh":[{"descriptor_ui":"D000072669","descriptor_name":"Gene Editing","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D000229","descriptor_name":"Dependovirus","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":true},{"descriptor_ui":"D000818","descriptor_name":"Animals","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006412","descriptor_name":"Hematopoietic Stem Cells","qualifier_ui":"Q000378","qualifier_name":"metabolism","is_major_topic":true},{"descriptor_ui":"D006412","descriptor_name":"Hematopoietic Stem Cells","qualifier_ui":"Q000821","qualifier_name":"virology","is_major_topic":true},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D007966","descriptor_name":"Leukodystrophy, Metachromatic","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D007966","descriptor_name":"Leukodystrophy, Metachromatic","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D010661","descriptor_name":"Phenylketonurias","qualifier_ui":"Q000235","qualifier_name":"genetics","is_major_topic":false},{"descriptor_ui":"D010661","descriptor_name":"Phenylketonurias","qualifier_ui":"Q000628","qualifier_name":"therapy","is_major_topic":false},{"descriptor_ui":"D014161","descriptor_name":"Transduction, Genetic","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D015316","descriptor_name":"Genetic Therapy","qualifier_ui":null,"qualifier_name":null,"is_major_topic":true},{"descriptor_ui":"D019076","descriptor_name":"Transgenes","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":3,"locations":[{"id":"doi:10.1089/hum.2020.049","doi":"https://doi.org/10.1089/hum.2020.049","is_oa":false,"landing_page_url":"https://doi.org/10.1089/hum.2020.049","pdf_url":null,"source":{"id":"https://openalex.org/S39730559","display_name":"Human Gene Therapy","issn_l":"1043-0342","issn":["1043-0342","1557-7422"],"is_oa":false,"is_in_doaj":false,"is_core":true,"listed_in":["anvur-scientific","cwts-core","dongbi-b","doyens","jufo-1","medline","norway-1","poland-100","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320443","host_organization_name":"Mary Ann Liebert, Inc.","host_organization_lineage":["https://openalex.org/P4310320443","https://openalex.org/P4310320017"],"host_organization_lineage_names":["Mary Ann Liebert, Inc.","SAGE Publishing"],"type":"journal"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Human Gene Therapy","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:7232699","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/7232699","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":"Hum Gene Ther","raw_type":"Text"},{"id":"pmid:32253938","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/32253938","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":"Human gene therapy","raw_type":"Journal Article"}],"best_oa_location":{"id":"pmh:oai:pubmedcentral.nih.gov:7232699","doi":null,"is_oa":true,"landing_page_url":"https://www.ncbi.nlm.nih.gov/pmc/articles/7232699","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":"Hum Gene Ther","raw_type":"Text"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","score":0.98580002784729,"display_name":"Good health and well-being"}],"study_designs":[],"awards":[{"id":"https://openalex.org/G5361983605","display_name":"Genetic Modification of Human Hematopoietic Stem Cells with Pseudotyped rAAV","funder_award_id":"R01HL087285","funder_id":"https://openalex.org/F4320337338","funder_display_name":"National Heart, Lung, and Blood Institute"},{"id":"https://openalex.org/G6558925553","display_name":"MICROBIAL GENOME CENTERS","funder_award_id":"n01ai40001-16-0-1","funder_id":"https://openalex.org/F4320332161","funder_display_name":"National Institutes of Health"},{"id":"https://openalex.org/G845353460","display_name":null,"funder_award_id":"N01-AI-40001","funder_id":"https://openalex.org/F4320337355","funder_display_name":"National Institute of Allergy and Infectious Diseases"},{"id":"https://openalex.org/G9000889907","display_name":"Cancer Center Support Grant","funder_award_id":"P30CA033572","funder_id":"https://openalex.org/F4320337351","funder_display_name":"National Cancer Institute"}],"funders":[{"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/F4320337355","display_name":"National Institute of Allergy and Infectious Diseases","ror":"https://ror.org/043z4tv69"}],"has_content":{"grobid_xml":false,"pdf":false},"content_urls":null,"referenced_works_count":75,"referenced_works":["https://openalex.org/W5003167","https://openalex.org/W6006485","https://openalex.org/W1484444210","https://openalex.org/W1496294057","https://openalex.org/W1526425334","https://openalex.org/W1526535705","https://openalex.org/W1569774431","https://openalex.org/W1622554753","https://openalex.org/W1644720114","https://openalex.org/W1748809707","https://openalex.org/W1824389368","https://openalex.org/W1842480026","https://openalex.org/W1901315342","https://openalex.org/W1906949314","https://openalex.org/W1909557371","https://openalex.org/W1945342395","https://openalex.org/W1967895335","https://openalex.org/W1968067596","https://openalex.org/W1968368792","https://openalex.org/W1968382713","https://openalex.org/W1968546180","https://openalex.org/W1970306935","https://openalex.org/W1975134688","https://openalex.org/W1985810133","https://openalex.org/W1993439119","https://openalex.org/W2009431650","https://openalex.org/W2010666698","https://openalex.org/W2014010760","https://openalex.org/W2016745966","https://openalex.org/W2017354518","https://openalex.org/W2019612944","https://openalex.org/W2023319852","https://openalex.org/W2027884666","https://openalex.org/W2028592334","https://openalex.org/W2030287652","https://openalex.org/W2040312123","https://openalex.org/W2045158993","https://openalex.org/W2045343535","https://openalex.org/W2047531087","https://openalex.org/W2057126633","https://openalex.org/W2057548071","https://openalex.org/W2063144816","https://openalex.org/W2067189573","https://openalex.org/W2073287658","https://openalex.org/W2083535960","https://openalex.org/W2084595762","https://openalex.org/W2088686424","https://openalex.org/W2090650873","https://openalex.org/W2091413140","https://openalex.org/W2097072158","https://openalex.org/W2112624632","https://openalex.org/W2115588616","https://openalex.org/W2122236774","https://openalex.org/W2136085939","https://openalex.org/W2143836588","https://openalex.org/W2152187442","https://openalex.org/W2153175573","https://openalex.org/W2154450422","https://openalex.org/W2158417626","https://openalex.org/W2172663840","https://openalex.org/W2255844952","https://openalex.org/W2414645227","https://openalex.org/W2437299318","https://openalex.org/W2534223396","https://openalex.org/W2736065260","https://openalex.org/W2793858050","https://openalex.org/W2884783182","https://openalex.org/W2953856578","https://openalex.org/W2955699452","https://openalex.org/W2972621860","https://openalex.org/W2972831855","https://openalex.org/W2990985577","https://openalex.org/W2994233443","https://openalex.org/W3010969896","https://openalex.org/W4249789152"],"related_works":["https://openalex.org/W2129283689","https://openalex.org/W2012134951","https://openalex.org/W2045343535","https://openalex.org/W2095054797","https://openalex.org/W2053384038","https://openalex.org/W1780966310","https://openalex.org/W2068392166","https://openalex.org/W118733061","https://openalex.org/W7096854529","https://openalex.org/W6926877895"],"abstract_inverted_index":{"Adeno-associated":[0],"virus":[1],"(AAV)-based":[2],"vectors":[3,33,75,150],"have":[4,98,102,114],"transformed":[5],"into":[6],"powerful":[7],"elements":[8],"of":[9,67,70,80,83,120,138,158,169,196,218],"genetic":[10,106],"medicine":[11],"with":[12,31,73],"proven":[13],"therapeutic":[14,176],"efficacy":[15],"and":[16,127,135,173,212],"a":[17,81,117,204],"good":[18],"safety":[19],"profile.":[20],"Over":[21],"the":[22,65,78,92,110,124,128,132,139,155,167,175,194,199,216],"years,":[23],"efforts":[24],"to":[25,47,77,101,160],"transduce":[26,136],"hematopoietic":[27,111],"stem":[28,95,200],"cells":[29,137],"(HSCs)":[30],"AAV2":[32,43,74],"have,":[34],"however,":[35],"been":[36],"challenging.":[37],"While":[38],"there":[39],"was":[40,58],"evidence":[41],"that":[42,190],"delivered":[44],"vector":[45],"genomes":[46],"primitive,":[48],"quiescent,":[49],"multipotential,":[50],"self-renewing,":[51],"in":[52,166,208],"vivo":[53,209],"engrafting":[54],"HSCs,":[55],"transgene":[56],"expression":[57],"elusive.":[59],"In":[60],"this":[61],"study,":[62],"we":[63],"review":[64,154],"evolution":[66],"AAV":[68],"transduction":[69],"HSC,":[71,91],"starting":[72],"leading":[76],"isolation":[79],"family":[82],"naturally":[84],"occurring":[85],"AAVs":[86],"from":[87,183],"human":[88],"CD34":[89],"+":[90],"AAVHSC.":[93],"The":[94],"cell-derived":[96,201],"AAVHSCs":[97,113,159],"turned":[99],"out":[100],"remarkable":[103],"potentials":[104],"for":[105,116,193,207,215],"therapies":[107],"well":[108],"beyond":[109],"system.":[112,142],"tropism":[115],"wide":[118],"variety":[119],"peripheral":[121],"tissues,":[122],"including":[123],"liver,":[125],"muscle,":[126],"retina.":[129],"They":[130],"cross":[131],"blood\u2013brain":[133],"barrier":[134],"central":[140],"nervous":[141],"Preclinical":[143],"gene":[144,187,210],"therapy":[145,188,211],"studies":[146],"underway":[147,192],"using":[148],"AAVHSC":[149],"are":[151],"discussed.":[152],"We":[153,178],"notable":[156],"ability":[157],"mediate":[161],"efficient,":[162],"seamless":[163],"homologous":[164],"recombination":[165],"absence":[168],"exogenous":[170],"nuclease":[171],"activity":[172],"discuss":[174,180],"implications.":[177],"also":[179],"early":[181],"results":[182],"an":[184],"AAVHSC-based":[185],"clinical":[186],"trial":[189],"is":[191],"treatment":[195,217],"phenylketonuria.":[197],"Thus,":[198],"AAVHSC,":[202],"offer":[203],"multifaceted":[205],"platform":[206],"genome":[213],"editing":[214],"inherited":[219],"diseases.":[220]},"counts_by_year":[{"year":2026,"cited_by_count":5},{"year":2025,"cited_by_count":2},{"year":2024,"cited_by_count":4},{"year":2023,"cited_by_count":1},{"year":2022,"cited_by_count":1},{"year":2021,"cited_by_count":3}],"updated_date":"2026-10-31T12:09:11.096373","created_date":"2020-04-10T00:00:00"}