In vivo genome editing of the albumin locus as a platform for protein replacement therapy
Author:
Affiliation:
1. Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA;
2. Howard Hughes Medical Institute, Philadelphia, PA; and
3. Sangamo BioSciences, Richmond, CA
Abstract
Publisher
American Society of Hematology
Subject
Cell Biology,Hematology,Immunology,Biochemistry
Link
http://ashpublications.org/blood/article-pdf/126/15/1777/1389342/1777.pdf
Reference27 articles.
1. Long-term safety and efficacy of factor IX gene therapy in hemophilia B.;Nathwani;N Engl J Med,2014
2. AAV2 gene therapy readministration in three adults with congenital blindness.;Bennett;Sci Transl Med,2012
3. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery.;Lombardo;Nat Biotechnol,2007
4. Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases.;Perez;Nat Biotechnol,2008
5. Targeted genome editing in human repopulating haematopoietic stem cells.;Genovese;Nature,2014
Cited by 245 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. AAV mediated gene therapy for haemophilia B: From the early attempts to modern trials;Thrombosis Research;2024-02
2. Genome editing technologies;Rigor and Reproducibility in Genetics and Genomics;2024
3. Regeneration and anti-inflammatory effects of stem cells and their extracellular vesicles in gynecological diseases;Biomedicine & Pharmacotherapy;2023-12
4. CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice;Molecular Therapy - Methods & Clinical Development;2023-12
5. Production of therapeutic levels of human FIX-R338L by engineered B cells using GMP-compatible medium;Molecular Therapy - Methods & Clinical Development;2023-12
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3