Related Experiment Video

Updated: Jul 4, 2025

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification
04:43

Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification

Published on: October 11, 2024

1.9K

The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

P J Brooks, Timothy M Miller, Frédéric Revah

    Nature Reviews. Drug Discovery
    |February 6, 2024
    PubMed
    Abstract

    No abstract available in PubMed .

    More Related Videos

    Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants09:20

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

    Published on: October 18, 2022

    4.5K
    Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models07:21

    Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models

    Published on: June 30, 2021

    6.1K

    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...
    25.4K