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Immunity|December 30, 2019
Targeted Complement Inhibition at Synapses Prevents Microglial Synaptic Engulfment and Synapse Loss in Demyelinating DiseaseSebastian Werneburg, Jonathan Jung, Rejani B Kunjamma, et al.
Neuropsychopharmacology : Official Publication of the American College of Neuropsychopharmacology|July 6, 2019
Conditional, inducible gene silencing in dopamine neurons reveals a sex-specific role for Rit2 GTPase in acute cocaine response and striatal functionCarolyn G Sweeney, Patrick J Kearney, Rita R Fagan, et al.
Nature Communications|July 2, 2020
Structural characterization of a novel human adeno-associated virus capsid with neurotropic propertiesHung-Lun Hsu, Alexander Brown, Anna B Loveland, et al.
Biorxiv : the Preprint Server for Biology|March 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.
EMBO Molecular Medicine|February 27, 2024
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotorQing Xie, Xiupeng Chen, Hong Ma, et al.
Biorxiv : the Preprint Server for Biology|March 30, 2023
Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing <i>in vivo</i>Han Zhang, Karen Kelly, Jonathan Lee, et al.
Gastroenterology|May 30, 2025
Adeno-associated Virus-mediated Silencing of Sox4 Leads to Long-Term Amelioration of Liver Phenotypes in Mouse Models of Alagille SyndromeDuncan Fox, Jun Xie, Jennifer L Burwinkel, et al.
Elife|June 8, 2021
Start codon disruption with CRISPR/Cas9 prevents murine Fuchs' endothelial corneal dystrophyHironori Uehara, Xiaohui Zhang, Felipe Pereira, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 12, 2022
Evaluating the state of the science for adeno-associated virus integration: An integrated perspectiveDenise E Sabatino, Frederic D Bushman, Randy J Chandler, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 14, 2024
In vivo evolution of env in SHIV-AD8<sub>EO</sub>-infected rhesus macaques after AAV-vectored delivery of eCD4-IgDaniel O'Hagan, Siddhartha Shandilya, Lincoln J Hopkins, et al.
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