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 therapy using bone marrow transplantation.

P M Lehn1

  • 1Whitehead Institute for Biomedical Research, Cambridge, MA 02142.

Bone Marrow Transplantation
|February 1, 1987
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

[Gene therapy in onco-hematology. Perspectives and ethics].

Soins; la revue de reference infirmiere·1990
Same author

Gene therapy using bone marrow transplantation: a 1990 update.

Bone marrow transplantation·1990
See all related articles

Gene transfer into bone marrow stem cells shows promise for treating genetic diseases. While in vivo expression remains a challenge, successful reconstitution in mice suggests future therapeutic potential.

Area of Science:

  • Molecular Biology
  • Hematology
  • Genetics

Background:

  • Advancements in molecular biology enable characterization and cloning of genes responsible for genetic diseases.
  • Replication-defective retroviruses serve as efficient vectors for gene transfer into various cell types.

Purpose of the Study:

  • To explore the potential of bone marrow as a target for gene transfer due to its self-renewing stem cells.
  • To assess the feasibility of gene incorporation and reconstitution of hematopoietic stem cells in a murine model.

Main Methods:

  • Utilizing retroviral vectors for gene transfer into hematopoietic stem cells.
  • Collecting, manipulating ex vivo, and reinfusing bone marrow cells.
  • Evaluating gene incorporation and hematopoietic reconstitution in syngeneic murine recipients.

Related Experiment Videos

Main Results:

  • Successful incorporation of genes into hematopoietic stem cells was demonstrated in a murine system.
  • Complete reconstitution of recipient bone marrow with modified stem cells was achieved.
  • In vivo gene expression following transfer has not yet been adequately achieved, though in vitro expression is documented.

Conclusions:

  • Bone marrow transplantation is a viable strategy for gene transfer, with potential for reconstituting hematopoietic stem cells.
  • Further improvements in retroviral vector design are needed to achieve appropriate in vivo gene expression.
  • Successful gene transfer and expression in bone marrow stem cells could offer novel therapeutic options for genetic disorders.