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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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Foilsithe in:Biomolecules
Main Authors: Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Formáid: Artigo
Teanga:Inglês
Foilsithe: MDPI 2021
Ábhair:
Rochtain Ar Líne: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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