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

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

690
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
690
Gene Therapy00:59

Gene Therapy

26.9K
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...
26.9K
What is Genetic Engineering?00:49

What is Genetic Engineering?

77.7K
Overview
77.7K

You might also read

Related Articles

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

Sort by
Same author

Economics achieved through the use of artificial intelligence-powered contouring solutions in a network of oncology clinics in low- and middle-income countries.

Frontiers in oncology·2026
Same author

Recent immunisation and anaesthesia: Is it a problem?

Journal of perioperative practice·2025
Same author

Comparison of Energy Consumption Between Single Energy O-Gantry and Dual Energy C-Arm Linear Accelerator.

Advances in radiation oncology·2025
Same author

Transition from point A to volume based image guided brachytherapy across a network of centers in India through workshop and mentoring.

Brachytherapy·2024
Same author

Pilot study of radiation oncology peer review in low middle income country (LMIC) through cloud-based platform.

Journal of cancer research and therapeutics·2024
Same author

Evaluation of a deep image-to-image network (DI2IN) auto-segmentation algorithm across a network of cancer centers.

Journal of cancer research and therapeutics·2024

Related Experiment Video

Updated: Nov 27, 2025

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

5.0K

Development of Gene Therapy Vectors: Remaining Challenges.

Vibhor Gupta1, Sílvia P Lourenço1, Ismael J Hidalgo1

  • 1Absorption Systems, LLC, Exton, PA 19341, USA.

Journal of Pharmaceutical Sciences
|December 6, 2020
PubMed
Summary

Gene therapy, using viral vectors like adeno-associated viruses (AAVs), shows promise for treating genetic disorders. However, challenges remain in AAV product development, including assessing neutralizing antibodies and ensuring in-vitro bio potency for clinical success.

Keywords:
Adeno-associated virus (AAV)Gene deliveryGene therapyNeutralizing antibody (Nab) assayPotency assay

More Related Videos

Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models
07:21

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

Published on: June 30, 2021

6.8K
Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications
03:52

Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

Published on: February 9, 2024

3.3K

Related Experiment Videos

Last Updated: Nov 27, 2025

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

5.0K
Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models
07:21

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

Published on: June 30, 2021

6.8K
Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications
03:52

Author Spotlight: Efficient Adeno-Associated Virus Isolation for Pre-Clinical Applications

Published on: February 9, 2024

3.3K

Area of Science:

  • Biotechnology
  • Gene Therapy
  • Viral Vectors

Background:

  • The approval of Luxturna® marks a significant advancement in gene therapy for inherited blindness, following early setbacks in the field.
  • Gene therapy strategies involve gene replacement or editing to restore protein function, utilizing various viral and non-viral delivery vectors.
  • Recombinant adeno-associated viruses (AAVs) are favored delivery vehicles due to their safety, specificity, and long-term stability, despite challenges.

Purpose of the Study:

  • To highlight critical aspects of adeno-associated virus (AAV) product development for gene therapy.
  • To discuss the ongoing challenges in AAV pre-clinical and clinical development.
  • To emphasize the importance of cell-based assays for AAV development.

Main Methods:

  • Review of current gene therapy approaches and vector technologies.
  • Analysis of challenges in AAV product development and regulatory pathways.
  • Discussion of cell-based assays for evaluating neutralizing antibodies and in-vitro bio potency.

Main Results:

  • Non-viral vectors offer alternatives to viral vectors by mitigating immunogenicity and neutralizing antibody issues.
  • AAVs have demonstrated early success in gene therapy delivery, but face hurdles in development and approval.
  • Engineering of AAV serotypes necessitates robust assays for assessing seroprevalence and bio potency.

Conclusions:

  • Addressing challenges in AAV development, including immunogenicity and potency assays, is crucial for advancing gene therapy.
  • Continued research into AAV vectors and associated assays will facilitate the successful translation of gene therapies into clinical practice.
  • Optimizing AAV delivery systems and characterization methods is key to overcoming current limitations in gene therapy product development.