AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study

Abstract excerpt

AAV-mediated gene therapy holds promise for the treatment of lysosomal storage diseases (LSDs), some of which are already in clinical trials. Yet, ultra-rare subtypes of LSDs, such as some glycoproteinoses, have lagged. Here, we report on a long-term safety and efficacy preclinical study conducted in the murine model of galactosialidosis, a glycoproteinosis caused by a deficiency of protective protein/cathepsin A...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Lysosomal Storage Disorders Research
• Share to Toxoplasma gondii Research Studies
• Share to Virus-based gene therapy research