Efficacy of dual intracerebroventricular and intravitreal CLN5 gene therapy in sheep prompts the first clinical trial to treat CLN5 Batten disease

• Main Authors: Samantha J. Murray (Author), Martin P. Wellby (Author), Graham K. Barrell (Author), Katharina N. Russell (Author), Ashley R. Deane (Author), John R. Wynyard (Author), Steven J. Gray (Author), David N. Palmer (Author), Nadia L. Mitchell (Author)
• Format: Book
• Published: Frontiers Media S.A., 2023-10-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mutations in the CLN5 gene cause the fatal, pediatric, neurodegenerative disease CLN5 neuronal ceroid lipofuscinosis. Affected children suffer progressive neuronal loss, visual failure and premature death. Presently there is no treatment. This study evaluated dual intracerebroventricular (ICV) and i...