- A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects. The study, conducted in nonhuman primates, is published in the peer-reviewed journal Human Gene Therapy. Click here to read the article now. CLN2 disease is a fatal, childhood autosomal recessive disorder cause by mutations in the CLN2 gene, which encodes tripeptidyl peptidase (TPP-1). In a prior study, the investigators, Ronald Crystal, MD, and Dolan Sondhi, PhD, from Weill Cornell Medical College, and coauthors, found that intraparenchymal administration of an adeno-associated virus (AAV) vector encoding human CLN2 slowed but did not stop disease progression.

https://www.miragenews.com/advances-in-gene-therapy-for-cln2-batten-disease-1094034/

#miragenews