Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease

CLN5 neuronal ceroid lipofuscinosis (NCL, Batten disease) is a rare, inherited fatal neurodegenerative disorder caused by mutations in the CLN5 gene. The disease is characterised by progressive neuronal loss, blindness, and premature death. There is no cure. This study evaluated the efficacy of intr...

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Detalles Bibliográficos
Autores principales: Mitchell, Nadia L., Murray, Samantha J., Wellby, Martin P., Barrell, Graham K., Russell, Katharina N., Deane, Ashley R., Wynyard, John R., Palmer, Madeleine J., Pulickan, Anila, Prendergast, Phillipa M., Casy, Widler, Gray, Steven J., Palmer, David N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10442658/
https://www.ncbi.nlm.nih.gov/pubmed/37614821
http://dx.doi.org/10.3389/fgene.2023.1212228

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