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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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Updated: Mar 27, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Current Progress in Therapeutic Gene Editing for Monogenic Diseases.

Versha Prakash1, Marc Moore1, Rafael J Yáñez-Muñoz1

  • 1School of Biological Sciences, Royal Holloway, University of London, Egham, Surrey, UK.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|January 15, 2016
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Summary

Programmable nucleases enable precise genome engineering for human gene therapy. This review covers nuclease-based treatments for inherited diseases like cystic fibrosis and hemophilia, discussing progress and challenges.

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Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Molecular Biology

Background:

  • Programmable nucleases offer precise genome alteration capabilities.
  • Genome engineering is crucial for research, biotechnology, and therapeutic applications.
  • Nuclease-based gene editing underpins novel human gene therapy strategies.

Purpose of the Study:

  • To review the current progress of nuclease-based therapeutic applications for inherited monogenic diseases.
  • To highlight the challenges and future prospects of these gene editing technologies.

Main Methods:

  • Review of current literature on nuclease-based gene editing.
  • Discussion of therapeutic strategies utilizing nonhomologous end joining and homology-dependent repair.
  • Focus on applications in cystic fibrosis, Duchenne muscular dystrophy, bone marrow diseases, and hemophilia.

Main Results:

  • Nuclease-based gene editing has advanced therapeutic strategies for monogenic diseases.
  • Significant progress has been made in applying these technologies to inherited conditions.
  • Key challenges and future directions in nuclease-based therapies are identified.

Conclusions:

  • Nuclease-based gene editing holds great promise for treating inherited monogenic diseases.
  • Continued research and development are essential to overcome associated challenges.
  • The future of gene therapy is significantly influenced by advancements in programmable nucleases.