In this study, we developed a novel mouse model of RP (huRhoP347S) expressing a pathogenic human rhodopsin gene with a Pro347Ser (P347S) mutation on a rhodopsin knockout background.

Christian Mueller and coauthors from Sanofi describe a state-of-the-art ML model that predicts the fitness of AAV2 capsid mutants based on the amino acid sequence of the capsid monomer.

Forbes Porter, from the Eunice Kennedy Shriver National Institute of Child Health and Human Development, and coauthors assessed the prevalence of anti-AAV9 and anti-AAV2 neutralizing antibodies ...

In the present study, we found that Stat3-wt and Stat3-ca were sufficient to activate axonal regeneration after an optic nerve injury. However, a 3D analysis revealed that the course of growing ...

MeiraGTx (MGTX) announced that the U.S. Food and Drug Administration, FDA, has granted Regenerative Medicine Advanced Therapy, RMAT, designation to AAV2-hAQP1 for the treatment of Grade 2/3 ...

The Phase 1 AQUAx clinical trial is an open-label, non-randomized, dose escalation trial designed to evaluate the safety of MeiraGTx’s investigational gene therapy AAV2-hAQP1 when administered ...

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies ...