Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics

BACKGROUND: Batten disease is characterized by cognitive and motor impairment, retinal degeneration, and seizures leading to premature death. Recent studies have shown efficacy for a gene therapy approach for CLN7 Batten disease. This gene therapy approach is promising to treat cognitive and motor i...

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Detalles Bibliográficos
Autores principales: Rowe, Ashley A., Chen, Xin, Nettesheim, Emily R., Issioui, Yacine, Dong, Thomas, Hu, Yuhui, Messahel, Souad, Kayani, Saima N., Gray, Steven J., Wert, Katherine J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9626557/
https://www.ncbi.nlm.nih.gov/pubmed/36374771
http://dx.doi.org/10.1016/j.ebiom.2022.104314

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9626557/
https://www.ncbi.nlm.nih.gov/pubmed/36374771
http://dx.doi.org/10.1016/j.ebiom.2022.104314

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