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 ...

We further conducted in vitro and in vivo functional delivery of mRNA and found that the LNP fraction with the highest mRNA loading levels was the least transfection competent.

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 ...

Lastly, daily IVIS images were taken to track and measure transfection over time. Discussion: These data together reveal that FMCM-LPX-FLuc could serve as a promising mRNA delivery platform for ...

Recent advances in ocular gene and cellular therapy rely on precisely controlled subretinal delivery. Due to its inherent limitations, manual delivery can lead to iatrogenic damage to the retina, the ...