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RD114-pseudotyped oncoretroviral vectors. Biological and physical properties
P F Kelly1, J Carrington, A Nathwani
1Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, 332 North Lauderdale, Memphis, TN 38105, USA.
Annals of the New York Academy of Sciences
|July 19, 2001
Summary
Feline endogenous virus (RD114) envelope proteins enable efficient gene transfer into human hematopoietic stem cells. This advance in oncoretroviral vector technology shows promise for clinical applications in gene therapy.
Area of Science:
- Molecular Biology
- Gene Therapy
- Stem Cell Research
Background:
- Limited viral envelope receptor expression hinders efficient oncoretroviral gene transfer.
- Developing effective gene transfer methods for human hematopoietic stem cells is crucial for therapeutic applications.
Purpose of the Study:
- To evaluate envelope proteins for enhanced gene transfer efficiency into primitive human hematopoietic stem cells.
- To identify oncoretroviral vectors suitable for clinical gene therapy applications.
Main Methods:
- Assessed gene transfer efficiency of various oncoretroviral vectors pseudotyped with different envelope proteins.
- Utilized primitive human hematopoietic stem cell populations for transduction experiments.
- Evaluated vector stability and concentration methods.
Main Results:
- Oncoretroviral vectors pseudotyped with feline endogenous virus (RD114) envelope efficiently transduced human repopulating cells after a single exposure.
- RD114-pseudotyped vectors demonstrated comparable in vitro stability to amphotropic vectors.
- RD114-pseudotyped vectors could be concentrated without compromising stem cell potential.
Conclusions:
- The RD114 envelope protein significantly enhances oncoretroviral gene transfer efficiency into human hematopoietic stem cells.
- RD114-pseudotyped vectors offer a promising platform for clinical gene therapy due to their efficiency and potential for concentration.
- Increased efficiency is likely due to higher receptor expression or affinity for the RD114 envelope.