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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: Jul 9, 2026

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

[Gene therapy using AAV].

Keiya Ozawa1

  • 1Division of Hematology, Department of Medicine, Center for Molecular Medicine, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke-shi, Tochigi 329-0498, Japan. kozawa@ms2.jichi.ac.jp

Uirusu
|November 28, 2007
PubMed
Summary

Adeno-associated virus (AAV) vectors show promise for gene therapy due to their safety and efficacy in delivering genes. Researchers are exploring AAV for treating Parkinson's disease, monogenic disorders, and cancer, with potential for site-specific integration in stem cells.

Area of Science:

  • Gene therapy
  • Viral vectors
  • Molecular biology

Context:

  • Adeno-associated virus (AAV) vectors are derived from non-pathogenic viruses.
  • AAV vectors efficiently transduce non-dividing cells and ensure long-term gene expression.
  • Specific AAV serotypes are selected based on target cell types.

Purpose:

  • To explore the therapeutic potential of adeno-associated virus (AAV) vectors in gene therapy.
  • To investigate AAV vector applications for neurological disorders like Parkinson's disease (PD).
  • To evaluate AAV for treating monogenic diseases, cancer, and for stem cell gene therapy.

Summary:

  • AAV vectors are promising for gene therapy, particularly for Parkinson's disease by enabling dopamine synthesis via AADC gene transfer.

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

Published on: October 11, 2024

Related Experiment Videos

Last Updated: Jul 9, 2026

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

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

  • Intramuscular AAV injection is suitable for protein-supplement gene therapy in monogenic diseases like hemophilia and Fabry disease.
  • AAV vectors can be used in cancer gene therapy to inhibit tumor growth and metastasis, and for site-specific integration in stem cells to minimize mutagenesis.

    • Impact:

    • AAV vectors offer a versatile platform for diverse gene therapy applications.
    • Potential to develop novel treatments for neurodegenerative diseases, genetic disorders, and cancer.
    • Advancements in site-specific integration enhance the safety and efficacy of stem cell-based therapies.