Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems.

Marina Beraza-MillorJulen Rodríguez-CastejónAna Del Pozo-RodríguezAlicia Rodríguez-GascónMaría Angeles Solinís Aspiazu

Published in: BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy (2024)

Nucleic acid therapies are expanding the clinical tools and therapies currently available for LSDs. Recent advances in CRISPR/Cas9 technology and the growing impact of nanotechnology are expected to boost the clinical translation of gSRT in the near future, and not only for LSDs.

Keyphrases

systematic review
nucleic acid
crispr cas
genome editing
stem cells
randomized controlled trial
gene expression
current status
mesenchymal stem cells
copy number
amino acid