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

Update on hematopoietic stem cell gene transfer using non-human primate models.

Jiong Hu1, Cynthia E Dunbar

  • 1Molecular Hematopoiesis Hematology Branch, National Heart Lung and Blood Institutes, National Institutes of Health, Bethesda, MD 20892, USA.

Current Opinion in Molecular Therapeutics
|November 19, 2002
PubMed
Summary

Preclinical gene transfer into hematopoietic stem cells (HSCs) in large animals is crucial for treating genetic diseases. Optimizing gene delivery and developing selection strategies are key for clinical success.

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

Small interfering RNA-mediated silencing of mutant NPM1 suppresses acute myeloid leukemia via reversing KAT7 and p300-mediated histone acetylation.

Leukemia·2026
Same author

Sequential CLAGE-Ven (cladribine, cytarabine, etoposide, and venetoclax) with reduced-intensity conditioning improves outcomes in patients with refractory acute myeloid leukemia: a prospective phase II study.

Bone marrow transplantation·2026
Same author

Microglial clonal dynamics and the impact of clonal hematopoiesis in autologously transplanted rhesus macaques.

Cell reports·2026
Same author

Restricting glycine uptake with bitopertin improves erythropoiesis in preclinical models of Diamond-Blackfan anemia.

Blood. Red cells & iron·2026
Same author

Soluble epoxide hydrolase drives neurovascular dysfunction in a model of amyloidosis.

Brain : a journal of neurology·2026
Same author

Clonal Selection and Evolution after Treatment of Severe Aplastic Anemia.

NEJM evidence·2026

Area of Science:

  • Hematology
  • Gene Therapy
  • Translational Medicine

Background:

  • Gene transfer into hematopoietic stem cells (HSCs) offers a promising therapeutic avenue for genetic and hematological disorders.
  • Large animal models, particularly non-human primates, are essential for preclinical evaluation due to their biological similarity to humans, surpassing murine models.
  • Current gene transfer studies in non-human primates primarily utilize standard retroviral vectors, with significant progress in efficiency.

Purpose of the Study:

  • To highlight the importance of preclinical gene transfer studies in large animal models for assessing the safety and effectiveness of gene therapies.
  • To review the current status of gene transfer techniques targeting HSCs in non-human primates.
  • To identify future directions for advancing gene therapy for hematological diseases.

Related Experiment Videos

Main Methods:

  • Review of existing literature on gene transfer into HSCs in large animal models.
  • Focus on retroviral vector applications and preliminary exploration of lentiviral and alternative vector systems.
  • Discussion of necessary advancements in post-transduction selection and expansion strategies.

Main Results:

  • Standard retroviral vectors have shown significant advances in gene transfer efficiency in non-human primate HSCs.
  • The use of lentiviral and other novel vector systems in large animal models is still in its nascent stages.
  • The need for improved post-transduction selection and/or expansion methods is evident for clinical translation.

Conclusions:

  • Preclinical gene transfer in large animals is vital for the clinical translation of HSC-based therapies.
  • Further research into optimizing gene transfer efficiency and exploring alternative vector systems is required.
  • Development of robust selection and expansion strategies is critical for the successful clinical application of gene therapy for hematological diseases.