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Related Experiment Videos

Gene therapy: basic concepts and recent advances

D L Sokol1, A M Gewirtz

  • 1Department of Pathology, University of Pennsylvania School of Medicine, Philadelphia 19104, USA.

Critical Reviews in Eukaryotic Gene Expression
|January 1, 1996
PubMed
Summary
This summary is machine-generated.

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Human gene therapy trials show promise but face challenges in effectiveness and efficiency. Ongoing research focuses on correcting genetic defects, enhancing cell traits, and improving gene transfer methods for therapeutic applications.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Human gene therapy clinical trials have been ongoing for approximately five years.
  • Despite preliminary modeling, achieving effective and efficient human gene therapy remains a significant challenge.
  • Over 100 gene therapy protocols have been submitted for approval, indicating growing interest in the field.

Purpose of the Study:

  • To review major developments in human gene therapy.
  • To discuss the impact of these developments on the progress of the field.
  • To highlight diverse goals of gene therapy protocols, including disease correction and cell engineering.

Main Methods:

  • Review of in vitro and in vivo modeling data.
  • Analysis of clinical trial outcomes and submitted protocols.

Related Experiment Videos

  • Investigation of various gene transfer methods for therapeutic nucleotide delivery.

    • Main Results:

    • Gene therapy aims to correct inherited defects, engineer new cellular traits (e.g., diminished chemosensitivity), and modify tumor cell immunophenotypes.
    • Cellular tracking in vitro and in vivo is a key goal, achieved by marking cells with detectable genes.
    • Research is actively exploring efficient gene transfer techniques to deliver therapeutic nucleotides.

    Conclusions:

    • Human gene therapy has progressed significantly, with numerous protocols submitted and diverse applications explored.
    • While challenges in efficacy and efficiency persist, ongoing research in gene transfer and therapeutic applications continues to advance the field.
    • The field shows a strong trajectory with continued interest and development in gene therapy strategies.