Advancement of potential gene therapy could offer new hope for children with Batten disease - Earth News Report
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. CLN2 disease is a fatal, childhood autosomal recessive disorder
