International journal of molecular sciences

Using CRISPR-Cas9 Gene Editing to Treat Inherited Liver Diseases

Updated

Abstract

CRISPR-Cas9 gene editing technology may offer transformative hope for treating hereditary liver diseases.

  • Inherited liver diseases are mostly caused by single-gene mutations.
  • Current treatment options for these conditions are largely non-curative.
  • Liver transplantation is complicated by organ shortages, immune rejection, and lifelong medication requirements.
  • Recent advancements in CRISPR-Cas9 technology could improve treatment strategies for these diseases.
  • The review outlines the principles and benefits of CRISPR-Cas9 and its advanced forms, including base and prime editing.
  • Challenges remain for the clinical application of these gene editing technologies.

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Funding

Competing interests

0 of 5
authors report competing interests
5 report none
PubMed

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