Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems
Author:
Funder
Ministerio de Ciencia, Innovación y Universidades
Eusko Jaurlaritza
Euskal Herriko Unibertsitatea
Universidad del País Vasco
Publisher
Springer Science and Business Media LLC
Link
https://link.springer.com/content/pdf/10.1007/s40259-024-00674-1.pdf
Reference202 articles.
1. Parenti G, Andria G, Ballabio A. Lysosomal storage diseases: from pathophysiology to therapy. Annu Rev Med. 2015;66:471–86.
2. Platt FM, d'Azzo A, Davidson BL, Neufeld EF, Tifft CJ. Lysosomal storage diseases. Nat Rev Dis Primers. 2018;4:27
3. Filocamo M, Morrone A. Lysosomal storage disorders: molecular basis and laboratory testing. Hum Genomics. 2011;5:156–69.
4. Arenz C. Recent advances and novel treatments for sphingolipidoses. Future Med Chem. 2017;9:1685–98.
5. Fecarotta S, Tarallo A, Damiano C, Minopoli N, Parenti G. Pathogenesis of mucopolysaccharidoses, an update. Int J Mol Sci. 2020;21:1–14.
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