Related Experiment Video
Updated: Jan 28, 2026

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
Published on: August 2, 2018
AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods
Brett Palaschak1, Roland W Herzog1,2, David M Markusic3
1Department of Pediatrics, University of Florida, Gainesville, FL, USA.
Abstract:
Adeno-associated virus (AAV) vectors to treat liver-specific genetic diseases are the focus of several ongoing clinical trials. The ability to give a peripheral injection of virus that will successfully target the liver is one of many attractive features of this technology. Although initial studies of AAV liver gene transfer revealed some limitations, extensive animal modeling and further clinical development have helped solve some of these issues, resulting in several successful clinical trials that have reached curative levels of clotting factor expression in hemophilia. Looking beyond gene replacement, recent technologies offer the possibility for AAV liver gene transfer to directly repair deficient genes and potentially treat autoimmune disease.
More Related Videos
09:20Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
Published on: October 18, 2022
21:55Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling
Published on: April 2, 2012
Related Concept Videos
Controlled-Current Coulometry: Overview
Drug Delivery: Overview
Enteral delivery involves administering drugs directly through swallowing, sublingual placement, or buccal application. Orally administered drugs predominantly navigate the...
Methods Of Healthcare Delivery System
Managed Care System:
The managed care system is designed to control the cost while maintaining the quality of care. The patient's care from admission to discharge is planned by the primary care provider or the case manager, also known as the gatekeeper. In a managed care system, the number of care providers is...
Sampling Methods: Overview
In analytical chemistry, the choice of...
Interfacial Electrochemical Methods: Overview
Cell Specific Gene Expression