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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Vydáno v:Biomolecules
Hlavní autoři: Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2021
Témata:
Review
On-line přístup: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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