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CLN7 gene therapy: hope for an ultra-rare condition
CLN7 Batten disease, also known as variant late infantile neuronal ceroid lipofuscinosis type 7 (vLINCL7), is an ultra-rare form of Batten disease that presents early in life with severe neurological symptoms, including visual deficits, motor problems, and frequent seizures. There is high unmet need...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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American Society for Clinical Investigation
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8884894/ https://www.ncbi.nlm.nih.gov/pubmed/35229731 http://dx.doi.org/10.1172/JCI157820 |