Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

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...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

[The safety and efficacy of adeno-associated virus-mediated LDLR transfection in homozygous familial hypercholesterolemia].

Zhonghua xin xue guan bing za zhi·2026
Same author

[Research progress on the physiological mechanism of external auditory canal self-cleaning].

Zhonghua er bi yan hou tou jing wai ke za zhi = Chinese journal of otorhinolaryngology head and neck surgery·2026
Same author

Abstracts of the 26th International Workshop on Clinical Pharmacology of HIV, Hepatitis and other Antiviral Drugs 2025, 3-4 September 2025, Amsterdam, the Netherlands.

British journal of clinical pharmacology·2025
Same author

Prognostic value of relative enhancement metrics based on preconcurrent chemoradiotherapy magnetic resonance imaging (pre-CCRT MRI) volumetric segmentation in glioblastoma.

Clinical radiology·2025
Same author

Arginine Dentifrices and Childhood Caries Prevention: A Randomized Clinical Trial.

JDR clinical and translational research·2025
Same author

[Oral submucosal fibrosis induced by active components in areca nut: a network pharmacology-based analysis and validation of the mechanism].

Nan fang yi ke da xue xue bao = Journal of Southern Medical University·2024

Related Experiment Video

Updated: Jun 27, 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

Tailoring the AAV vector capsid for gene therapy.

L H Vandenberghe1, J M Wilson, G Gao

  • 1Gene Therapy Program, Division of Transfusion Medicine, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA.

Gene Therapy
|December 5, 2008
PubMed
Summary

Adeno-associated virus (AAV) shows promise for gene therapy, but host-vector complexities hinder clinical translation. Researchers are exploring AAV capsid variants, rational design, and directed evolution to improve vector functionality for better gene transfer.

More Related Videos

Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
09:21

Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors

Published on: January 29, 2019

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling
21:55

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

Published on: April 2, 2012

Related Experiment Videos

Last Updated: Jun 27, 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

Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
09:21

Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors

Published on: January 29, 2019

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling
21:55

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

Published on: April 2, 2012

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Gene Therapy

Background:

  • Adeno-associated virus (AAV) is a highly efficient vector for gene therapy, with demonstrated in vivo gene transfer capabilities.
  • Translating AAV technology to clinical applications is challenging due to complex host-vector interactions across different species.

Purpose of the Study:

  • To explore strategies for overcoming challenges in adeno-associated virus (AAV) gene therapy vector development.
  • To enhance the functionality of AAV vectors for improved clinical translation.

Main Methods:

  • Characterization of naturally occurring mammalian adeno-associated virus (AAV) capsid variants.
  • Rational design of second-generation AAV vectors based on structural and functional knowledge.
  • Application of directed evolution to isolate improved AAV vectors under selective laboratory pressures.

Main Results:

  • Gained structural and functional insights into natural AAV capsid variants.
  • Developed a series of second-generation AAV vectors with improved properties through rational design.
  • Identified novel AAV vectors with enhanced functionality via directed evolution.

Conclusions:

  • Exploring AAV capsid variations is crucial for advancing gene therapy vector technology.
  • Rational design and directed evolution represent promising strategies for optimizing AAV vectors.
  • Improved AAV vectors hold significant potential for overcoming clinical translation barriers in gene therapy.