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Gene Therapy Leaves a Vicious Cycle

Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1

  • 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States.

Frontiers in Oncology
|May 10, 2019
PubMed

Insights

Gene therapy offers a one-time fix for genetic disorders by correcting errors in DNA. Recent advances in gene editing and viral vectors are revitalizing this "living drug" approach for various diseases.

Area of Science:

  • Genetics
  • Molecular Biology
  • Biotechnology

Background:

  • Genetic mutations cause diseases, and current treatments are limited and often cause side effects.
  • Gene therapy, a "living drug," aims to correct genetic errors for a one-time cure.
  • Viral vector-based gene therapy has faced skepticism but is regaining interest.

Purpose of the Study:

  • To review viral vectors used in gene therapy.
  • To discuss advances and challenges in gene therapy.
  • To explore future perspectives for gene therapy.

Main Methods:

  • Review of current literature on gene therapy.
  • Analysis of viral vector technologies.
  • Examination of gene editing advancements.

Main Results:

  • Gene therapy offers a potential cure for genetic disorders by correcting DNA errors.
  • Advancements in gene editing and viral vectors have renewed interest in gene therapy.
  • Over 12 gene therapy drugs are now available for conditions like cancer and blindness.

Conclusions:

  • Gene therapy is a promising one-time treatment for genetic diseases.
  • Continued research in viral vectors and gene editing is crucial.
  • Gene therapy holds significant potential for treating a wide range of disorders.
Keywords:
clinical trialsdiseases and disordersgene therapymodern medicinesviral vectors

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