Targeted retroviral transduction of c-kit+ hematopoietic cells using novel ligand display technology

Gene therapy for a wide variety of disorders would be greatly enhanced by the development of vectors that could be targeted for gene delivery to specific populations of cells. We describe here high-efficiency targeted transduction based on a novel targeting strategy that exploits the ability of retr...

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Bibliographic Details
Published in:Blood 2004-11, Vol.104 (9), p.2697-2703
Main Authors: Chandrashekran, Anil, Gordon, Myrtle Y., Casimir, Colin
Format: Article
Language:English
Subjects:
Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy
Applied cell therapy and gene therapy
Biological and medical sciences
Capsid Proteins
Cell Line, Tumor
Drug Delivery Systems - methods
Green Fluorescent Proteins - genetics
Hematopoietic Stem Cells - chemistry
Hematopoietic Stem Cells - metabolism
Humans
Ligands
Medical sciences
Protein Engineering
Proto-Oncogene Proteins c-kit
Retroviridae - genetics
Stem Cell Factor
Transduction, Genetic - methods
Transfusions. Complications. Transfusion reactions. Cell and gene therapy
Virus Assembly
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Summary:Gene therapy for a wide variety of disorders would be greatly enhanced by the development of vectors that could be targeted for gene delivery to specific populations of cells. We describe here high-efficiency targeted transduction based on a novel targeting strategy that exploits the ability of retroviruses to incorporate host cell proteins into the surface of the viral particle as they bud through the plasma membrane. Ecotropic retroviral particles produced in cells engineered to express the membrane-bound form of stem cell factor (mbSCF) transduce both human cell lines and primary cells with high efficiency in a strictly c-kit (SCF receptor)-dependent fashion. The availability of efficient targeted vectors provides a platform for the development of a new generation of therapies using in vivo gene delivery. (Blood. 2004;104: 2697-2703)
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2003-10-3717