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

Gene transfer into hematopoietic stem cells

A W Nienhuis1

  • 1Clinical Hematology Branch, National Heart, Lung and Blood Institute, Bethesda, Maryland.

Blood Cells
|January 1, 1994
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

Lentiviral vectors for the treatment of Wiskott-Aldrich syndrome.

Gene therapy·2008
Same author

Current status and prospects for gene therapy.

Vox sanguinis·2004
Same author

Functional correction of T cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein.

Gene therapy·2003
Same author

Bone marrow-derived cells contribute to tumor neovasculature and, when modified to express an angiogenesis inhibitor, can restrict tumor growth in mice.

Clinical cancer research : an official journal of the American Association for Cancer Research·2001
Same author

Autocrine expression of both endostatin and green fluorescent protein provides a synergistic antitumor effect in a murine neuroblastoma model.

Cancer gene therapy·2001
Same author

Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy.

Blood·2001
Same journal

In vitro and in vivo studies of stromal niches.

Blood cells·1994
Same journal

Differential manipulation of normal and chronic myeloid leukemia stem cell proliferation in vitro.

Blood cells·1994
Same journal

Enrichment for primitive hemopoietic progenitors of marrow cells from 5-fluorouracil-treated mice and normal mice.

Blood cells·1994
Same journal

Protein tyrosine phosphorylation in the regulation of hematopoiesis by receptors of the cytokine-receptor superfamily.

Blood cells·1994
Same journal

Lysis of blood platelets by human osteosarcoma cells in vitro.

Blood cells·1994
Same journal

Development of hemopoietic bone marrow within the ectopic bone induced by bone morphogenetic protein.

Blood cells·1994
See all related articles

Gene transfer into hematopoietic stem cells shows promise for treating genetic diseases. Retroviral vectors enable gene insertion, but efficiency improvements are needed for broader therapeutic applications.

Area of Science:

  • * Hematology
  • * Gene Therapy
  • * Molecular Biology

Background:

  • * Gene therapy holds potential for treating genetic and acquired diseases.
  • * Retroviral vectors are being explored for gene insertion into hematopoietic stem cells (HSCs).
  • * Successful gene transfer into HSCs could enable lineage-specific gene expression in hematopoietic organs post-bone marrow transplantation.

Purpose of the Study:

  • * To investigate the efficacy of retroviral vectors for gene transfer into HSCs.
  • * To assess the potential of genetically modified HSCs for long-term reconstitution.
  • * To evaluate gene transfer efficiency in different animal models and human cells.

Main Methods:

  • * Retroviral vector-mediated gene transfer into HSCs in murine and rhesus monkey models.

Related Experiment Videos

  • * Mobilization of peripheral blood stem cells using cytokine administration.
  • * Genetic marking of human bone marrow and peripheral blood cells.
  • * Assessment of long-term reconstitution and gene expression in transplant recipients.

    • Main Results:

    • * In murine models, 20-30% of repopulating stem cells were genetically modified.
    • * Peripheral blood stem cells were efficiently transduced and capable of long-term reconstitution.
    • * In rhesus monkeys, long-term repopulation was achieved, but vector genome frequency was low (1-2%).
    • * Human cell marking demonstrated potential for autologous transplantation.

    Conclusions:

    • * Retroviral vector-mediated gene transfer is feasible for HSCs, enabling long-term reconstitution.
    • * Current vector genome frequencies require improvement for widespread therapeutic use.
    • * Future research will focus on enhancing gene transfer efficiencies for clinical applications.