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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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Gene therapy: progress and predictions.

Mary Collins1, Adrian Thrasher2

  • 1Division of Infection and Immunity, University College London, Gower Street, London WC1E 6BT, UK Division of Advanced Therapies, National Institute for Biological Standards and Control, Blanche Lane, Potters Bar, Herts EN6 3QG, UK mary.collins@ucl.ac.uk.

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Summary
This summary is machine-generated.

Gene therapy has advanced significantly over 25 years, offering treatments for inherited diseases and cancer. Future gene therapy will improve safety, efficacy, and incorporate gene editing for broader applications.

Keywords:
evolutionary medicinegene therapypersonalized medicine

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Medical Science

Background:

  • The first clinical gene delivery trial occurred 25 years ago, marking a milestone in medical science.
  • Gene therapy has evolved from marker gene insertion to therapeutic applications for various diseases.

Purpose of the Study:

  • To review the progress of gene therapy over the past 25 years.
  • To highlight current and future prospects of gene therapy, including gene editing.

Main Methods:

  • Review of clinical gene delivery advancements.
  • Analysis of current gene therapy applications in inherited disorders, cancer, and infectious diseases.

Main Results:

  • Successful gene therapy approaches now exist for certain inherited single-gene defects, inherited retinopathies, and hemophilia B.
  • Promising gene therapy strategies are emerging for cancer and infectious diseases.

Conclusions:

  • Gene therapy has demonstrated significant progress and therapeutic potential.
  • Future gene therapy will focus on enhanced safety, efficacy, manufacturing, and the integration of gene editing technologies.
  • Gene delivery may offer a cost-effective method for biological medicine delivery.