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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Detalles Bibliográficos
Autores principales: Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8074255/
https://www.ncbi.nlm.nih.gov/pubmed/33924076
http://dx.doi.org/10.3390/biom11040611

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8074255/
https://www.ncbi.nlm.nih.gov/pubmed/33924076
http://dx.doi.org/10.3390/biom11040611

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