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

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...

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Detalles Bibliográficos
Autores principales: Murray, Samantha J., Wellby, Martin P., Barrell, Graham K., Russell, Katharina N., Deane, Ashley R., Wynyard, John R., Gray, Steven J., Palmer, David N., Mitchell, Nadia L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Pharmacology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10628725/
https://www.ncbi.nlm.nih.gov/pubmed/37942487
http://dx.doi.org/10.3389/fphar.2023.1212235

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10628725/
https://www.ncbi.nlm.nih.gov/pubmed/37942487
http://dx.doi.org/10.3389/fphar.2023.1212235

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