Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques
Although clinical benefits have been reported in several human hematopoietic gene therapy trials, a remaining important goal is the transition to nonmyeloablative pretransplantation conditioning to decrease toxicity. Previous attempts at reduced intensity conditioning in nonhuman primates have resul...
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| Veröffentlicht in: | Human gene therapy 2012-07, Vol.23 (7), p.754-768 |
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| creator | VERHOEYEN, Els RELOUZAT, Francis DUBART-KUPPERSCHMITT, Anne PROST, Stephane CAMBOT, Marie COSTA, Caroline NEGRE, Didier LEGRAND, Faézeh JOUBERT, Christophe LE GRAND, Roger COSSET, François-Loïc LEBOULCH, Philippe |
| description | Although clinical benefits have been reported in several human hematopoietic gene therapy trials, a remaining important goal is the transition to nonmyeloablative pretransplantation conditioning to decrease toxicity. Previous attempts at reduced intensity conditioning in nonhuman primates have resulted in only temporary vector marking of autologous blood cells or their persistence at low levels, well below the thresholds for clinical efficacy. In addition, we reasoned that lentiviral vector particles displaying cytokines at their surface have the potential to preserve stem cell fitness better than current ex vivo transduction protocols, which involve exposure to cytokine overstimulation. Here we show that the classically nonmyeloablative agent fludarabine (30 mg/m(2)/day for 3 days) together with low-level total body irradiation (2 Gy) and the use of a stem cell factor-displaying simian immunodeficiency virus-based vector, resulted in sustained, single-copy vector marking of autologous blood cells in two macaques over 3 years posttransplantation at levels averaging 1% of all lineages. This percentage is within the range of anticipated efficacy levels for hemophilia and related diseases and forms a basis for further improvement. |
| doi_str_mv | 10.1089/hum.2012.020 |
| format | Article |
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Previous attempts at reduced intensity conditioning in nonhuman primates have resulted in only temporary vector marking of autologous blood cells or their persistence at low levels, well below the thresholds for clinical efficacy. In addition, we reasoned that lentiviral vector particles displaying cytokines at their surface have the potential to preserve stem cell fitness better than current ex vivo transduction protocols, which involve exposure to cytokine overstimulation. Here we show that the classically nonmyeloablative agent fludarabine (30 mg/m(2)/day for 3 days) together with low-level total body irradiation (2 Gy) and the use of a stem cell factor-displaying simian immunodeficiency virus-based vector, resulted in sustained, single-copy vector marking of autologous blood cells in two macaques over 3 years posttransplantation at levels averaging 1% of all lineages. This percentage is within the range of anticipated efficacy levels for hemophilia and related diseases and forms a basis for further improvement.</description><identifier>ISSN: 1043-0342</identifier><identifier>EISSN: 1557-7422</identifier><identifier>DOI: 10.1089/hum.2012.020</identifier><identifier>PMID: 22463386</identifier><identifier>CODEN: HGTHE3</identifier><language>eng</language><publisher>Larchmont, NY: Liebert</publisher><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Animals ; Antigens, CD34 - metabolism ; Applied cell therapy and gene therapy ; Autografts ; Biological and medical sciences ; Biotechnology ; Blood cells ; Chimerism ; Clinical trials ; Cytokines ; Fitness ; fludarabine ; Fundamental and applied biological sciences. Psychology ; Gene therapy ; Genetic Therapy ; Genetic Vectors ; Green Fluorescent Proteins - biosynthesis ; Green Fluorescent Proteins - genetics ; Health. Pharmaceutical industry ; HEK293 Cells ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells - metabolism ; Hemophilia ; Hemophilia A - therapy ; Hemopoiesis ; Humans ; Immunodeficiency ; Industrial applications and implications. Economical aspects ; Lymphocyte Count ; Macaca ; Macaca fascicularis ; Male ; Medical sciences ; Myeloablative Agonists - administration & dosage ; Primates ; Radiation ; Radiation-Sensitizing Agents - administration & dosage ; Recombinant Proteins - biosynthesis ; Recombinant Proteins - genetics ; Simian Immunodeficiency Virus - genetics ; Stem Cell Factor - genetics ; Stem cells ; Toxicity ; Transduction, Genetic ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Transplantation Conditioning ; Transplantation, Autologous ; Vidarabine - administration & dosage ; Vidarabine - analogs & derivatives ; Whole-Body Irradiation</subject><ispartof>Human gene therapy, 2012-07, Vol.23 (7), p.754-768</ispartof><rights>2014 INIST-CNRS</rights><rights>Copyright 2012, Mary Ann Liebert, Inc. 2012</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c447t-bfa06a6ff8b9a2a415741dccf9ca9dd6df48c72fab7d8c1f01be979c37d28d6e3</citedby><cites>FETCH-LOGICAL-c447t-bfa06a6ff8b9a2a415741dccf9ca9dd6df48c72fab7d8c1f01be979c37d28d6e3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,315,781,785,886,27929,27930</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=26679951$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/22463386$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>VERHOEYEN, Els</creatorcontrib><creatorcontrib>RELOUZAT, Francis</creatorcontrib><creatorcontrib>DUBART-KUPPERSCHMITT, Anne</creatorcontrib><creatorcontrib>PROST, Stephane</creatorcontrib><creatorcontrib>CAMBOT, Marie</creatorcontrib><creatorcontrib>COSTA, Caroline</creatorcontrib><creatorcontrib>NEGRE, Didier</creatorcontrib><creatorcontrib>LEGRAND, Faézeh</creatorcontrib><creatorcontrib>JOUBERT, Christophe</creatorcontrib><creatorcontrib>LE GRAND, Roger</creatorcontrib><creatorcontrib>COSSET, François-Loïc</creatorcontrib><creatorcontrib>LEBOULCH, Philippe</creatorcontrib><title>Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques</title><title>Human gene therapy</title><addtitle>Hum Gene Ther</addtitle><description>Although clinical benefits have been reported in several human hematopoietic gene therapy trials, a remaining important goal is the transition to nonmyeloablative pretransplantation conditioning to decrease toxicity. Previous attempts at reduced intensity conditioning in nonhuman primates have resulted in only temporary vector marking of autologous blood cells or their persistence at low levels, well below the thresholds for clinical efficacy. In addition, we reasoned that lentiviral vector particles displaying cytokines at their surface have the potential to preserve stem cell fitness better than current ex vivo transduction protocols, which involve exposure to cytokine overstimulation. Here we show that the classically nonmyeloablative agent fludarabine (30 mg/m(2)/day for 3 days) together with low-level total body irradiation (2 Gy) and the use of a stem cell factor-displaying simian immunodeficiency virus-based vector, resulted in sustained, single-copy vector marking of autologous blood cells in two macaques over 3 years posttransplantation at levels averaging 1% of all lineages. This percentage is within the range of anticipated efficacy levels for hemophilia and related diseases and forms a basis for further improvement.</description><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Animals</subject><subject>Antigens, CD34 - metabolism</subject><subject>Applied cell therapy and gene therapy</subject><subject>Autografts</subject><subject>Biological and medical sciences</subject><subject>Biotechnology</subject><subject>Blood cells</subject><subject>Chimerism</subject><subject>Clinical trials</subject><subject>Cytokines</subject><subject>Fitness</subject><subject>fludarabine</subject><subject>Fundamental and applied biological sciences. Psychology</subject><subject>Gene therapy</subject><subject>Genetic Therapy</subject><subject>Genetic Vectors</subject><subject>Green Fluorescent Proteins - biosynthesis</subject><subject>Green Fluorescent Proteins - genetics</subject><subject>Health. Pharmaceutical industry</subject><subject>HEK293 Cells</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Hematopoietic Stem Cells - metabolism</subject><subject>Hemophilia</subject><subject>Hemophilia A - therapy</subject><subject>Hemopoiesis</subject><subject>Humans</subject><subject>Immunodeficiency</subject><subject>Industrial applications and implications. Economical aspects</subject><subject>Lymphocyte Count</subject><subject>Macaca</subject><subject>Macaca fascicularis</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Myeloablative Agonists - administration & dosage</subject><subject>Primates</subject><subject>Radiation</subject><subject>Radiation-Sensitizing Agents - administration & dosage</subject><subject>Recombinant Proteins - biosynthesis</subject><subject>Recombinant Proteins - genetics</subject><subject>Simian Immunodeficiency Virus - genetics</subject><subject>Stem Cell Factor - genetics</subject><subject>Stem cells</subject><subject>Toxicity</subject><subject>Transduction, Genetic</subject><subject>Transfusions. Complications. Transfusion reactions. Cell and gene therapy</subject><subject>Transplantation Conditioning</subject><subject>Transplantation, Autologous</subject><subject>Vidarabine - administration & dosage</subject><subject>Vidarabine - analogs & derivatives</subject><subject>Whole-Body Irradiation</subject><issn>1043-0342</issn><issn>1557-7422</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2012</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNpVkUmPEzEQhVsIxCxw44x8QeIwHWy3e7sgRWE2KSMkJszVqnjpGNp2sN2M8hP5VziaEOBkS-_Tq1f1iuINwTOCu_7DZrIzigmdYYqfFaekrtuyZZQ-z3_MqhJXjJ4UZzF-w5hUddO-LE4oZU1Vdc1p8es-KYsWahzRFYjkQ_nJxO0IO-MGdG-sAYdurZ2cl0obYZQTO_RgAozoQe35iFZ-UGmjAno0aYMALf0jWngnTTLe7W2-qMFY5dB8HLOkfciIG8qVChZduiGATllOyGt0rZwq7yB8VxLNp-RHP_gpohtlIfmtNyoZgY6RIzIO3YGAH5OKr4oXGsaoXh_e8-Lr1eVqcVMuP1_fLubLUjDWpnKtATfQaN2te6DASN0yIoXQvYBeykZq1omWali3shNEY7JWfduLqpW0k42qzouPT77baW2VFDl5vgbfBmMh7LgHw_9XnNnwwf_kFcOM0TobvD8YBL8Pnrg1UeR1wKm8LCe46uq-r3uW0YsnVAQfY1D6OIZgvm-f5_b5vn2e28_423-jHeE_dWfg3QGAKGDUAZww8S_XNG0eTKrfAOe-sg</recordid><startdate>20120701</startdate><enddate>20120701</enddate><creator>VERHOEYEN, Els</creator><creator>RELOUZAT, Francis</creator><creator>DUBART-KUPPERSCHMITT, Anne</creator><creator>PROST, Stephane</creator><creator>CAMBOT, Marie</creator><creator>COSTA, Caroline</creator><creator>NEGRE, Didier</creator><creator>LEGRAND, Faézeh</creator><creator>JOUBERT, Christophe</creator><creator>LE GRAND, Roger</creator><creator>COSSET, François-Loïc</creator><creator>LEBOULCH, Philippe</creator><general>Liebert</general><general>Mary Ann Liebert, Inc</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7QO</scope><scope>7T5</scope><scope>7U9</scope><scope>8FD</scope><scope>FR3</scope><scope>H94</scope><scope>P64</scope><scope>RC3</scope><scope>5PM</scope></search><sort><creationdate>20120701</creationdate><title>Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques</title><author>VERHOEYEN, Els ; RELOUZAT, Francis ; DUBART-KUPPERSCHMITT, Anne ; PROST, Stephane ; CAMBOT, Marie ; COSTA, Caroline ; NEGRE, Didier ; LEGRAND, Faézeh ; JOUBERT, Christophe ; LE GRAND, Roger ; COSSET, François-Loïc ; LEBOULCH, Philippe</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c447t-bfa06a6ff8b9a2a415741dccf9ca9dd6df48c72fab7d8c1f01be979c37d28d6e3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2012</creationdate><topic>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</topic><topic>Animals</topic><topic>Antigens, CD34 - metabolism</topic><topic>Applied cell therapy and gene therapy</topic><topic>Autografts</topic><topic>Biological and medical sciences</topic><topic>Biotechnology</topic><topic>Blood cells</topic><topic>Chimerism</topic><topic>Clinical trials</topic><topic>Cytokines</topic><topic>Fitness</topic><topic>fludarabine</topic><topic>Fundamental and applied biological sciences. Psychology</topic><topic>Gene therapy</topic><topic>Genetic Therapy</topic><topic>Genetic Vectors</topic><topic>Green Fluorescent Proteins - biosynthesis</topic><topic>Green Fluorescent Proteins - genetics</topic><topic>Health. Pharmaceutical industry</topic><topic>HEK293 Cells</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Hematopoietic Stem Cells - metabolism</topic><topic>Hemophilia</topic><topic>Hemophilia A - therapy</topic><topic>Hemopoiesis</topic><topic>Humans</topic><topic>Immunodeficiency</topic><topic>Industrial applications and implications. Economical aspects</topic><topic>Lymphocyte Count</topic><topic>Macaca</topic><topic>Macaca fascicularis</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Myeloablative Agonists - administration & dosage</topic><topic>Primates</topic><topic>Radiation</topic><topic>Radiation-Sensitizing Agents - administration & dosage</topic><topic>Recombinant Proteins - biosynthesis</topic><topic>Recombinant Proteins - genetics</topic><topic>Simian Immunodeficiency Virus - genetics</topic><topic>Stem Cell Factor - genetics</topic><topic>Stem cells</topic><topic>Toxicity</topic><topic>Transduction, Genetic</topic><topic>Transfusions. Complications. Transfusion reactions. Cell and gene therapy</topic><topic>Transplantation Conditioning</topic><topic>Transplantation, Autologous</topic><topic>Vidarabine - administration & dosage</topic><topic>Vidarabine - analogs & derivatives</topic><topic>Whole-Body Irradiation</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>VERHOEYEN, Els</creatorcontrib><creatorcontrib>RELOUZAT, Francis</creatorcontrib><creatorcontrib>DUBART-KUPPERSCHMITT, Anne</creatorcontrib><creatorcontrib>PROST, Stephane</creatorcontrib><creatorcontrib>CAMBOT, Marie</creatorcontrib><creatorcontrib>COSTA, Caroline</creatorcontrib><creatorcontrib>NEGRE, Didier</creatorcontrib><creatorcontrib>LEGRAND, Faézeh</creatorcontrib><creatorcontrib>JOUBERT, Christophe</creatorcontrib><creatorcontrib>LE GRAND, Roger</creatorcontrib><creatorcontrib>COSSET, François-Loïc</creatorcontrib><creatorcontrib>LEBOULCH, Philippe</creatorcontrib><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Biotechnology Research Abstracts</collection><collection>Immunology Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Human gene therapy</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>VERHOEYEN, Els</au><au>RELOUZAT, Francis</au><au>DUBART-KUPPERSCHMITT, Anne</au><au>PROST, Stephane</au><au>CAMBOT, Marie</au><au>COSTA, Caroline</au><au>NEGRE, Didier</au><au>LEGRAND, Faézeh</au><au>JOUBERT, Christophe</au><au>LE GRAND, Roger</au><au>COSSET, François-Loïc</au><au>LEBOULCH, Philippe</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques</atitle><jtitle>Human gene therapy</jtitle><addtitle>Hum Gene Ther</addtitle><date>2012-07-01</date><risdate>2012</risdate><volume>23</volume><issue>7</issue><spage>754</spage><epage>768</epage><pages>754-768</pages><issn>1043-0342</issn><eissn>1557-7422</eissn><coden>HGTHE3</coden><abstract>Although clinical benefits have been reported in several human hematopoietic gene therapy trials, a remaining important goal is the transition to nonmyeloablative pretransplantation conditioning to decrease toxicity. Previous attempts at reduced intensity conditioning in nonhuman primates have resulted in only temporary vector marking of autologous blood cells or their persistence at low levels, well below the thresholds for clinical efficacy. In addition, we reasoned that lentiviral vector particles displaying cytokines at their surface have the potential to preserve stem cell fitness better than current ex vivo transduction protocols, which involve exposure to cytokine overstimulation. Here we show that the classically nonmyeloablative agent fludarabine (30 mg/m(2)/day for 3 days) together with low-level total body irradiation (2 Gy) and the use of a stem cell factor-displaying simian immunodeficiency virus-based vector, resulted in sustained, single-copy vector marking of autologous blood cells in two macaques over 3 years posttransplantation at levels averaging 1% of all lineages. This percentage is within the range of anticipated efficacy levels for hemophilia and related diseases and forms a basis for further improvement.</abstract><cop>Larchmont, NY</cop><pub>Liebert</pub><pmid>22463386</pmid><doi>10.1089/hum.2012.020</doi><tpages>15</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 1043-0342 |
| ispartof | Human gene therapy, 2012-07, Vol.23 (7), p.754-768 |
| issn | 1043-0342 1557-7422 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3404425 |
| source | MEDLINE; Alma/SFX Local Collection |
| subjects | Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Animals Antigens, CD34 - metabolism Applied cell therapy and gene therapy Autografts Biological and medical sciences Biotechnology Blood cells Chimerism Clinical trials Cytokines Fitness fludarabine Fundamental and applied biological sciences. Psychology Gene therapy Genetic Therapy Genetic Vectors Green Fluorescent Proteins - biosynthesis Green Fluorescent Proteins - genetics Health. Pharmaceutical industry HEK293 Cells Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells - metabolism Hemophilia Hemophilia A - therapy Hemopoiesis Humans Immunodeficiency Industrial applications and implications. Economical aspects Lymphocyte Count Macaca Macaca fascicularis Male Medical sciences Myeloablative Agonists - administration & dosage Primates Radiation Radiation-Sensitizing Agents - administration & dosage Recombinant Proteins - biosynthesis Recombinant Proteins - genetics Simian Immunodeficiency Virus - genetics Stem Cell Factor - genetics Stem cells Toxicity Transduction, Genetic Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transplantation Conditioning Transplantation, Autologous Vidarabine - administration & dosage Vidarabine - analogs & derivatives Whole-Body Irradiation |
| title | Stem Cell Factor-Displaying Simian Immunodeficiency Viral Vectors Together with a Low Conditioning Regimen Allow for Long-Term Engraftment of Gene-Marked Autologous Hematopoietic Stem Cells in Macaques |
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