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Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
CLN3 Batten disease is an autosomal recessive, neurodegenerative, lysosomal storage disease caused by mutations in CLN3, which encodes a lysosomal membrane protein(1–3). There are no disease-modifying treatments for this disease that affects up to 1 in 25,000 births, has an onset of symptoms in earl...
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| Publicado no: | Nat Med |
|---|---|
| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8008709/ https://ncbi.nlm.nih.gov/pubmed/32719489 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-020-0986-1 |
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