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 Experiment Videos

Human embryonic stem cells and gene therapy.

Yael Strulovici1, Philip L Leopold, Timothy P O'Connor

  • 1Department of Genetic Medicine, Weill Medical College of Cornell University, New York, NY 10021, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|March 16, 2007
PubMed
Summary
This summary is machine-generated.

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia: A Nonrandomized Clinical Trial.

JAMA cardiology·2026
Same author

Impact of population-specific pharmacogenomic variants on drug dosing in ICU patients.

The pharmacogenomics journal·2026
Same author

Consequences of human genetic variations in <i>KIAA0319L</i>, encoding adeno-associated virus receptor, on AAV-mediated gene transfer.

Molecular therapy. Advances·2026
Same author

Smoking-induced dysregulation of transposable elements in the small airway epithelium.

Respiratory research·2026
Same author

Prime Editing of Alzheimer's Disease High-Risk APOE4 Allele by Brain-Directed Adeno-Associated Virus Vectors.

Human gene therapy·2025
Same author

Genes required for <i>Mycobacterium tuberculosis</i> to survive the transition from aerosol to pulmonary alveolar lining fluid and early infection in a model of transmission.

bioRxiv : the preprint server for biology·2025

Gene therapy combined with human embryonic stem cells (hESCs) offers a promising approach for regenerative medicine. This review explores gene transfer strategies to overcome challenges in hESC differentiation and therapeutic application.

Area of Science:

  • Regenerative Medicine
  • Stem Cell Biology
  • Gene Therapy

Background:

  • Human embryonic stem cells (hESCs) offer a potential source for engineered tissues to restore function in diseased organs.
  • Significant challenges remain in controlling hESC differentiation, managing cell proliferation post-transplantation, and avoiding immune rejection of hESC-derived cells.

Purpose of the Study:

  • To present a framework for combined gene transfer and hESC therapies.
  • To review current knowledge on hESC derivation, characterization, differentiation, and available cell lines.
  • To analyze the application and challenges of gene transfer technologies in hESC-based therapeutics.

Main Methods:

  • Review of scientific literature on hESC derivation, differentiation protocols, and gene transfer techniques (viral and non-viral vectors).

Related Experiment Videos

  • Summarization of characteristics of approximately 500 reported hESC lines.
  • Analysis of gene transfer successes and failures in hESCs.

    • Main Results:

    • Gene therapy strategies can address critical hurdles in hESC therapeutic applications, including differentiation control, growth regulation, and immune evasion.
    • Gene transfer facilitates the isolation and expansion of genetically modified hESCs and can mitigate adverse effects.
    • Various viral and non-viral vectors have been explored for gene delivery to hESCs with mixed success.

    Conclusions:

    • Combined gene transfer and hESC therapies hold significant promise for regenerative medicine.
    • Overcoming challenges in controlled differentiation, safety, and immunogenicity is crucial for clinical translation.
    • Further research is needed to optimize gene transfer methods and fully realize the therapeutic potential of hESCs.