Previous efforts to circumvent the problem of the size limitation imposed by the AAV vector have focused mainly on altering the candidate genes into ‘mini-expression cassettes’ suitable for ...

we have demonstrated an increase in transgene expression of more than 600-fold from a minimal simian virus 40 (SV40) promoter and an increase in expression of 200-fold from the ITR in a vector ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...

A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...

Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

Solid Biosciences reported recent positive data in developing their gene therapy candidate SGT-003. The phase 1/2 INSPIRE ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

Nanoneedle technology offers the capability to simultaneously quantify titers of true, full viral genomes and a range of partial species.

The research team designed a nanomachine equipped with adeno-associated virus vectors (AAV ... applied as a new therapeutic approach to viral vector treatments, which are currently limited ...

Researchers from Tikkunlev Therapeutics Inc. and the University of Utah have presented preclinical data on TLT-101, a gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector ...