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CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments

8 May 2024

Abstract excerpt

In recent years, clustered regularly interspaced short palindromic repeats (CRISPRs) and CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat inherited disorders affecting different organ systems, such as blood and muscles. Both hematological and neuromuscular genetic disorders benefit from genome editing approaches but face different challenges in their clinical translation....

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PMID: 38786024
DOI: 10.3390/cells13100800

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