Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad brain distribution ...

Researchers have developed 'tomoseqr' -- a new software tool that enables easy estimation of the three-dimensional (3D) spatial distribution of gene expression. Tomoseqr is free to use and has been ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...