Related Experiment Video

Updated: Jul 9, 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

2.0K

Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated

John Puetz1

  • 1Department of Pediatrics, Division of Pediatric Hematology/Oncology, Saint Louis University School of Medicine, St. Louis, MO, United States.

Frontiers in Medicine
|November 29, 2023
PubMed

Keywords:
adeno-associated virusfactor IXfactor VIIIgene therapyhemophiliahepatocellular carcinomathrombotic microangiopathy

More Related Videos

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
Author Spotlight: Optimizing Digital Droplet PCR Method for Accurate Adeno-Associated Viral Genome Quantification04:43

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

Published on: October 11, 2024

2.0K

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