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Nature Biotechnology|February 2, 2016
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivoHao Yin, Chun-Qing Song, Joseph R Dorkin, et al.
Nature Genetics|May 26, 2025
Base editing of trinucleotide repeats that cause Huntington's disease and Friedreich's ataxia reduces somatic repeat expansions in patient cells and in miceZaneta Matuszek, Mandana Arbab, Maheswaran Kesavan, et al.
Nature Communications|January 21, 2017
Brain microvasculature defects and Glut1 deficiency syndrome averted by early repletion of the glucose transporter-1 proteinMaoxue Tang, Guangping Gao, Carlos B Rueda, et al.
Biorxiv : the Preprint Server for Biology|June 12, 2025
Gene therapies alleviate absence epilepsy associated with <i>Scn2a</i> deficiency in DBA/2J miceZaiyang Zhang, Jingliang Zhang, Xiaoling Chen, et al.
Nature Communications|June 6, 2023
Durable contraception in the female domestic cat using viral-vectored delivery of a feline anti-Müllerian hormone transgeneLindsey M Vansandt, Marie-Charlotte Meinsohn, Philippe Godin, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 17, 2015
Activation of Cyclic Adenosine Monophosphate Pathway Increases the Sensitivity of Cancer Cells to the Oncolytic Virus M1Kai Li, Haipeng Zhang, Jianguang Qiu, et al.
Human Gene Therapy|December 7, 2017
Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's DiseaseEdith L Pfister, Natalie DiNardo, Erica Mondo, et al.
Molecular Therapy. Nucleic Acids|June 12, 2025
AAVone: A cost-effective, single-plasmid solution for efficient AAV production with reduced DNA impuritiesRongze Yang, Ngoc Tam Tran, Taylor Chen, et al.
Genome Biology|December 18, 2019
Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouseJian-Ping Zhang, Xin-Xin Cheng, Mei Zhao, et al.
JCI Insight|May 22, 2019
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunityYuanyuan Xiao, Manish Muhuri, Shaoyong Li, et al.
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