Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells. 2019

Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center.

Hematopoietic stem and progenitor cell (HSPC) transplantation has been a cornerstone therapy for leukemia and other cancers for nearly half a century, underlies the only known cure of human immunodeficiency virus (HIV-1) infection, and shows immense promise in the treatment of genetic diseases such as beta thalassemia. Our group has developed a protocol to model HSPC gene therapy in nonhuman primates (NHPs), allowing scientists to optimize many of the same reagents and techniques that are applied in the clinic. Here, we describe methods for purifying CD34+ HSPCs and long-term persisting hematopoietic stem cell (HSC) subsets from primed bone marrow (BM). Identical techniques can be employed for the purification of other HSPC sources (e.g., mobilized peripheral blood stem cells [PBSCs]). Outlined is a 2 day protocol in which cells are purified, cultured, modified with lentivirus (LV), and prepared for infusion back into the autologous host. Key readouts of success include the purity of the CD34+ HSPC population, the ability of purified HSPCs to form morphologically distinct colonies in semisolid media, and, most importantly, gene modification efficiency. The key advantage to HSPC gene therapy is the ability to provide a source of long-lived cells that give rise to all hematopoietic cell types. As such, these methods have been used to model therapies for cancer, genetic diseases, and infectious diseases. In each case, therapeutic efficacy is established by enhancing the function of distinct HSPC progeny, including red blood cells, T cells, B cells, and/or myeloid subsets. The methods to isolate, modify, and prepare HSPC products are directly applicable and translatable to multiple diseases in human patients.

UI MeSH Term Description Entries
D011323 Primates An order of mammals consisting of more than 300 species that include LEMURS; LORISIDAE; TARSIERS; MONKEYS; and HOMINIDS. They are characterized by a relatively large brain when compared with other terrestrial mammals, forward-facing eyes, the presence of a CALCARINE SULCUS, and specialized MECHANORECEPTORS in the hands and feet which allow the perception of light touch. Primate
D000818 Animals Unicellular or multicellular, heterotrophic organisms, that have sensation and the power of voluntary movement. Under the older five kingdom paradigm, Animalia was one of the kingdoms. Under the modern three domain model, Animalia represents one of the many groups in the domain EUKARYOTA. Animal,Metazoa,Animalia
D015316 Genetic Therapy Techniques and strategies which include the use of coding sequences and other conventional or radical means to transform or modify cells for the purpose of treating or reversing disease conditions. Gene Therapy,Somatic Gene Therapy,DNA Therapy,Gene Therapy, Somatic,Genetic Therapy, Gametic,Genetic Therapy, Somatic,Therapy, DNA,Therapy, Gene,Therapy, Somatic Gene,Gametic Genetic Therapies,Gametic Genetic Therapy,Genetic Therapies,Genetic Therapies, Gametic,Genetic Therapies, Somatic,Somatic Genetic Therapies,Somatic Genetic Therapy,Therapies, Gametic Genetic,Therapies, Genetic,Therapies, Somatic Genetic,Therapy, Gametic Genetic,Therapy, Genetic,Therapy, Somatic Genetic
D018380 Hematopoietic Stem Cell Transplantation Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms. Stem Cell Transplantation, Hematopoietic,Transplantation, Hematopoietic Stem Cell
D019172 Transplantation Conditioning Preparative treatment of transplant recipient with various conditioning regimens including radiation, immune sera, chemotherapy, and/or immunosuppressive agents, prior to transplantation. Transplantation conditioning is very common before bone marrow transplantation. Conditioning, Transplantation,Conditionings, Transplantation,Transplantation Conditionings

Related Publications

Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
January 2023, Methods in molecular biology (Clifton, N.J.),
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
January 2023, Methods in molecular biology (Clifton, N.J.),
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
February 2019, Experimental hematology,
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
January 2001, Stem cells (Dayton, Ohio),
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
September 2012, Blood,
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
September 2003, Proceedings of the National Academy of Sciences of the United States of America,
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
September 1996, Human gene therapy,
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
January 2023, Methods in molecular biology (Clifton, N.J.),
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
September 2005, Experimental hematology,
Stefan Radtke, and Anai M Perez, and Rasika Venkataraman, and Sowmya Reddy, and Kevin G Haworth, and Olivier Humbert, and Hans-Peter Kiem, and Christopher W Peterson
August 2010, Current protocols in stem cell biology,
Copied contents to your clipboard!