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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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[Research progress in gene therapy for Wilson's disease].

S Tang1, L Bai2, S J Zheng1

  • 1The First Unit, Department of Hepatology, Beijing YouAn Hospital, Capital Medical University, Beijing 100069, China.

Zhonghua Gan Zang Bing Za Zhi = Zhonghua Ganzangbing Zazhi = Chinese Journal of Hepatology
|February 5, 2021
PubMed
Summary

Wilson's disease (WD) gene therapy offers a potential permanent cure for copper metabolism disorders. Research focuses on gene therapy vectors and CRISPR/Cas9 gene editing to overcome limitations of current WD treatments.

Keywords:
ATP7BGene therapyWilson disease

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Area of Science:

  • Genetics
  • Hepatology
  • Biotechnology

Background:

  • Wilson's disease (WD) is an inherited autosomal recessive disorder caused by ATP7B gene mutations, leading to impaired copper excretion.
  • Current treatments include drug therapy and liver transplantation, but face challenges like adherence, side effects, and donor scarcity.
  • Gene therapy presents a promising avenue for a permanent correction of copper metabolism in WD.

Purpose of the Study:

  • To review the current research progress in gene therapy for Wilson's disease.
  • To summarize advancements in gene therapy vectors suitable for WD treatment.
  • To discuss the application of the CRISPR/Cas9 gene editing system in addressing WD.

Main Methods:

  • Literature review of studies on Wilson's disease gene therapy.
  • Analysis of research on various gene therapy vectors (e.g., viral, non-viral).
  • Examination of CRISPR/Cas9 gene editing strategies targeting the ATP7B gene.

Main Results:

  • Gene therapy vectors are being developed to deliver functional ATP7B genes to liver cells.
  • CRISPR/Cas9 technology shows potential for precise correction of ATP7B mutations.
  • These approaches aim to restore normal copper metabolism and overcome treatment limitations.

Conclusions:

  • Gene therapy, particularly using advanced vectors and CRISPR/Cas9, holds significant potential for a curative treatment of Wilson's disease.
  • Further research and clinical trials are necessary to translate these findings into safe and effective therapies.
  • Gene editing offers a future direction for permanently managing copper overload in WD patients.