CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9

CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9

Lysosomal storage disorders (LSDs) are inherited metabolic conditions characterized by lysosomal enzyme deficiencies leading to substrate accumulation. As genetic diseases, LSDs can be treated with gene therapies (GT), including the CRISPR/Cas systems. The CRISPR/Cas systems enable precise and progr...

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Bibliographic Details
Main Authors: Andrés Felipe Leal, Luis Eduardo Prieto, Harry Pachajoa
Format: Article
Language:English
Published: MDPI AG 2025-07-01
Series:Cells
Subjects:
CRISPR/Cas
ex vivo
gene therapy
lysosomal storage disorders
Online Access:https://www.mdpi.com/2073-4409/14/15/1147
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