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Molecular Therapy. Advances|May 18, 2026
Engineering novel AAV capsids by broadly attenuated and subsequent muscle-specific tropism in mice and NHPsYue Pan, Yujian Zhong, Huan Chen, et al.
Nature Biotechnology|August 14, 2018
Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in miceDan Wang, Jia Li, Chun-Qing Song, et al.
Biorxiv : the Preprint Server for Biology|April 3, 2026
Postnatal gene restoration in succinic semialdehyde dehydrogenase deficiency (SSADHD) reveals phenotype reversibilityHenry H C Lee, Gabrielle McGinty, Amanda Liebhardt, et al.
Plos Pathogens|September 17, 2025
A single amino acid variant in the variable region I of AAV capsid confers liver detargetingRuxiao Xing, Mengyao Xu, Darcy Reil, et al.
The CRISPR Journal|December 18, 2023
A Fluorescent Reporter Mouse for <i>In Vivo</i> Assessment of Genome Editing with Diverse Cas Nucleases and Prime EditorsZexiang Chen, Suet-Yan Kwan, Aamir Mir, et al.
Science Immunology|February 28, 2025
Adeno-associated viral delivery of Env-specific antibodies prevents SIV rebound after discontinuing antiretroviral therapyVadim A Klenchin, Natasha M Clark, Nida K Keles, et al.
Biorxiv : the Preprint Server for Biology|June 19, 2024
Adeno-associated viral delivery of Env-specific antibodies prevents SIV rebound after discontinuing antiretroviral therapyVadim A Klenchin, Natasha M Clark, Nida K Keles, et al.
Human Gene Therapy|May 20, 2024
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus MonkeysShun-Qing Liang, Andrew W Navia, Michelle Ramseier, et al.
Nucleic Acids Research|November 30, 2023
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivoHan Zhang, Karen Kelly, Jonathan Lee, et al.
Proceedings of the National Academy of Sciences of the United States of America|May 13, 2021
Single-cell sequencing reveals suppressive transcriptional programs regulated by MIS/AMH in neonatal ovariesMarie-Charlotte Meinsohn, Hatice D Saatcioglu, Lina Wei, et al.
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