Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

Related Experiment Video

Updated: Aug 26, 2025

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
07:43

Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices

Published on: July 14, 2023

2.3K

Johannes Oldenburg1

  • 1University Clinic Bonn, Bonn, Germany johannes.oldenburg@ukbonn.de.

The New England Journal of Medicine
|October 5, 2022
PubMed
Abstract

No abstract available in PubMed .

More Related Videos

A Step-By-Step Method to Detect Neutralizing Antibodies Against AAV using a Colorimetric Cell-Based Assay05:53

A Step-By-Step Method to Detect Neutralizing Antibodies Against AAV using a Colorimetric Cell-Based Assay

Published on: December 7, 2021

4.7K
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

6.2K

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...
25.7K