Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. The suprachiasmatic nucleus (SCN) synchronises ...

an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ...

Zhang et al. present important findings that reveal a new role for TET2 in controlling glucose production in the liver, showing that both fasting and a high-fat diet increase TET2 levels, while its ...

LCA5 is a rare genetic disorder without any approved therapies to date. OPGx-LCA5 employs an adeno-associated virus vector (AAV8) to deliver a functional LCA5 gene to the retina. The investigational ...

GENV-HEM (AAV8.FVa) is a gene therapy for all hemophilias, including inhibitor patients, and without the durability issues that have plagued Factor VIII gene therapies. GENV-002 (AAV9.GAA and AAV9 ...

A second pediatric patient was recently dosed, and the pediatric cohort is expected to complete enrollment in Q2 2025; initial data from all three patients anticipated in Q3 2025 FDA Type D ...