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Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
Rare monogenic disorders such as lysosomal diseases have been at the forefront in the development of novel treatments where therapeutic options are either limited or unavailable. The increasing number of successful pre-clinical and clinical studies in the last decade demonstrates that gene therapy r...
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| Publicado no: | Biomolecules |
|---|---|
| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8074255/ https://ncbi.nlm.nih.gov/pubmed/33924076 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/biom11040611 |
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