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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Publicat a:Biomolecules
Autors principals: Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2021
Matèries:
Review
Accés en línia: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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