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...

Volledige beschrijving

Bewaard in:
Bibliografische gegevens
Gepubliceerd in:Biomolecules
Hoofdauteurs: Massaro, Giulia, Geard, Amy F., Liu, Wenfei, Coombe-Tennant, Oliver, Waddington, Simon N., Baruteau, Julien, Gissen, Paul, Rahim, Ahad A.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: MDPI 2021
Onderwerpen:
Review
Online toegang: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
Tags: Voeg label toe
Geen labels, Wees de eerste die dit record labelt!

Gelijkaardige items