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Targeting the Root Cause of Mucopolysaccharidosis IIIA with a New scAAV9 Gene Replacement Vector
No treatment is available to address the unmet needs of mucopolysaccharidosis (MPS) IIIA patients. Targeting the root cause, we developed a new self-complementary adeno-associated virus 9 (scAAV9) vector to deliver the human N-sulfoglucosamine sulfohydrolase (hSGSH) gene driven by a miniature cytome...
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| Publicado no: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7704409/ https://ncbi.nlm.nih.gov/pubmed/33313335 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.10.014 |
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