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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...

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Related Experiment Video

Updated: Jun 29, 2026

Quantification of Adeno-Associated Viral Genomes in Purified Vector Samples by Digital Droplet Polymerase Chain Reaction
04:43

Quantification of Adeno-Associated Viral Genomes in Purified Vector Samples by Digital Droplet Polymerase Chain Reaction

Published on: October 11, 2024

Gene therapy using adeno-associated virus vectors.

Shyam Daya1, Kenneth I Berns

  • 1Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida 32610-3610, USA.

Clinical Microbiology Reviews
|October 16, 2008
PubMed
Summary

Adeno-associated virus (AAV) is a promising gene therapy vector due to its unique life cycle and safety profile. Further research into AAV biology and vector development is crucial for clinical success.

Area of Science:

  • * Gene therapy vector development
  • * Virology and molecular biology

Background:

  • * Adeno-associated virus (AAV) possesses a unique life cycle enabling infection of both dividing and nondividing cells.
  • * Wild-type AAV exhibits no apparent pathogenicity, making it an attractive candidate for gene transfer.
  • * Gene transfer studies using AAV have demonstrated significant progress in animal models.

Purpose of the Study:

  • * To review current AAV biology and its application in gene therapy.
  • * To discuss the progress and safety of AAV vectors in clinical trials.
  • * To highlight novel approaches and recent findings for expanding AAV utility.

Main Methods:

  • * Comprehensive review of existing literature on AAV biology.
  • * Analysis of data from AAV-based gene transfer studies in animal models.

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Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling
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Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

Published on: April 2, 2012

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants
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Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants

Published on: October 18, 2022

Related Experiment Videos

Last Updated: Jun 29, 2026

Quantification of Adeno-Associated Viral Genomes in Purified Vector Samples by Digital Droplet Polymerase Chain Reaction
04:43

Quantification of Adeno-Associated Viral Genomes in Purified Vector Samples by Digital Droplet Polymerase Chain Reaction

Published on: October 11, 2024

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling
21:55

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

Published on: April 2, 2012

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants
09:20

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus (AAV) Capsid Variants

Published on: October 18, 2022

  • * Examination of clinical trial outcomes and safety data for AAV vectors.

    • Main Results:

    • * AAV vectors have shown notable safety in clinical trials.
    • * While proven efficacy is pending, some studies show promising observations.
    • * Significant progress has been made in preclinical AAV gene transfer studies.

    Conclusions:

    • * Deeper understanding of AAV biology is essential for advancing gene therapy.
    • * Development of more efficient AAV vectors is a key requirement for clinical efficacy.
    • * Combining ex vivo gene therapy with stem cell advancements offers future potential.