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

Transactivator and structurally optimized inducible lentiviral vectors.

Karin Haack1, Adam S Cockrell, Hong Ma

  • 1Gene Therapy Center, University of North Carolina at Chapel Hill, 7119 Thurston Bowles, CB 7352, Chapel Hill, NC 27599-7352, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|September 1, 2004
PubMed
Summary

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

Author Correction: Disease exacerbation in human DMD MYOrganoids enables gene therapy evaluation and unveils persistence of fibrotic activity.

NPJ Regenerative medicine·2026
Same author

Muscle meets Lysosomes: emerging strategies in muscular dystrophy.

Autophagy·2026
Same author

Disease exacerbation in human DMD MYOrganoids enables gene therapy evaluation and unveils persistence of fibrotic activity.

NPJ Regenerative medicine·2026
Same author

Lysosomal damage is a therapeutic target in Duchenne muscular dystrophy.

Science advances·2025
Same author

Envelope-dimer epitope 1 (EDE1) antibody (C10) treatment significantly reduces Zika virus replication in the male and female reproductive tracts.

Journal of virology·2025
Same author

Delivery of a Muscle-Targeted Adeno-Associated Vector Via <i>Ex Vivo</i> Normothermic Perfusion Is Efficient, Durable, and Safe in a Preclinical Porcine Heart Transplant Model.

Transplant international : official journal of the European Society for Organ Transplantation·2025

Researchers optimized lentiviral vectors for gene delivery. They developed an HIV-1 based system with improved doxycycline-dependent transgene expression for gene therapy and gene function analysis.

Area of Science:

  • Gene Therapy
  • Molecular Biology
  • Virology

Background:

  • Lentiviral vectors are valuable for gene delivery in research and therapy.
  • Controlling transgene expression is crucial for effective gene therapy and gene function studies.

Purpose of the Study:

  • To optimize HIV-1-based lentiviral vector systems for enhanced doxycycline-dependent transgene expression.
  • To reduce basal transgene expression and improve control over gene delivery both in vitro and in vivo.

Main Methods:

  • Modified HIV-1 vectors to dissect mechanisms of basal transgene expression.
  • Developed a binary vector system and relocated the inducible promoter to the U3 region of the LTR.
  • Substituted the VP16 transactivating domain with human p65 to enhance doxycycline-dependent expression.

Related Experiment Videos

Main Results:

  • Identified mechanisms contributing to doxycycline-independent transgene expression, including CMV promoter activity and translational readthrough.
  • Achieved significant reduction in basal activity by implementing a binary vector system and promoter relocation.
  • Demonstrated improved doxycycline-dependent transgene expression in various cell types and successful in vivo gene delivery in rat brains.

Conclusions:

  • Optimized HIV-1-based lentiviral vectors provide a powerful "toolbox" for controlled gene delivery.
  • The developed system enables precise control over transgene expression for applications in gene therapy and functional genomics.