High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors
Identifieur interne : 00CB97 ( Main/Merge ); précédent : 00CB96; suivant : 00CB98High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors
Auteurs : Francois Moreau-Gaudry [États-Unis, France, Canada, Australie, Italie, Taïwan] ; PING XIA ; GANG JIANG ; Natalya P. Perelman ; Gerhard Bauer ; James Ellis ; Katherine H. Surinya ; Fulvio Mavilio ; Che-Kun Shen ; Punam MalikSource :
- Blood [ 0006-4971 ] ; 2001.
Descripteurs français
- Pascal (Inist)
- Wicri :
- topic : Homme.
English descriptors
- KwdEn :
Abstract
Use of oncoretroviral vectors in gene therapy for hemoglobinopathies has been impeded by low titer vectors, genetic instability, and poor expression. Fifteen self-inactivating (SIN) lentiviral vectors using 4 erythroid promoters in combination with 4 erythroid enhancers with or without the woodchuck hepatitis virus postregulatory element (WPRE) were generated using the enhanced green fluorescent protein as a reporter gene. Vectors with high erythroid-specific expression in cell lines were tested in primary human CD34+ cells and in vivo in the murine bone marrow (BM) transplantation model. Vectors containing the ankyrin-1 promoter showed high-level expression and stable proviral transmission. Two vectors containing the ankyrin-1 promoter and 2 erythroid enhancers (HS-40 plus GATA-1 or HS-40 plus 5-aminolevulinate synthase intron 8 [18] enhancers) and WPRE expressed at levels higher than the HS2/β-promoter vector in bulk unilineage erythroid cultures and individual erythroid blast-forming units derived from human BM CD34+ cells. Sca1+/lineage- Ly5.1 mouse hematopoietic cells, transduced with these 2 ankyrin-1 promoter vectors, were injected into lethally irradiated Ly5.2 recipients. Eleven weeks after transplantation, high-level expression was seen from both vectors in blood (63%-89% of red blood cells) and erythroid cells in BM (70%-86% engraftment), compared with negligible expression in myeloid and lymphoid lineages in blood, BM, spleen, and thymus (0%-4%). The I8/HS-40-containing vector encoding a hybrid human [3/y-globin gene led to 43% to 113% human γ-globin expression/copy of the mouse α-globin gene. Thus, modular use of erythroid-specific enhancers/promoters and WPRE in SIN-lentiviral vectors led to identification of high-titer, stably transmitted vectors with high-level erythroid-specific expression for gene therapy of red cell diseases.
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Perelman</name></author><author><name sortKey="Bauer, Gerhard" sort="Bauer, Gerhard" uniqKey="Bauer G" first="Gerhard" last="Bauer">Gerhard Bauer</name></author><author><name sortKey="Ellis, James" sort="Ellis, James" uniqKey="Ellis J" first="James" last="Ellis">James Ellis</name></author><author><name sortKey="Surinya, Katherine H" sort="Surinya, Katherine H" uniqKey="Surinya K" first="Katherine H." last="Surinya">Katherine H. Surinya</name></author><author><name sortKey="Mavilio, Fulvio" sort="Mavilio, Fulvio" uniqKey="Mavilio F" first="Fulvio" last="Mavilio">Fulvio Mavilio</name></author><author><name sortKey="Shen, Che Kun" sort="Shen, Che Kun" uniqKey="Shen C" first="Che-Kun" last="Shen">Che-Kun Shen</name></author><author><name sortKey="Malik, Punam" sort="Malik, Punam" uniqKey="Malik P" first="Punam" last="Malik">Punam Malik</name></author></analytic><series><title level="j" type="main">Blood</title><title level="j" type="abbreviated">Blood</title><idno type="ISSN">0006-4971</idno><imprint><date when="2001">2001</date></imprint></series></biblStruct></sourceDesc><seriesStmt><title level="j" type="main">Blood</title><title level="j" type="abbreviated">Blood</title><idno type="ISSN">0006-4971</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Animal</term><term>Ankyrin</term><term>Enhancer sequence</term><term>Erythroid cell</term><term>Gene</term><term>Gene therapy</term><term>Genetic transfer</term><term>Globin</term><term>Hematopoietic cell</term><term>Hemoglobinopathy</term><term>Human</term><term>Lentivirus</term><term>Mouse</term><term>Transcription promoter</term><term>Treatment</term><term>Vector</term><term>Wood chuck hepatitis</term></keywords><keywords scheme="Pascal" xml:lang="fr"><term>Hémoglobinopathie</term><term>Traitement</term><term>Thérapie génique</term><term>Transfert génétique</term><term>Vecteur</term><term>Lentivirus</term><term>Hépatite marmotte</term><term>Promoteur transcription</term><term>Séquence activatrice</term><term>Cellule hématopoïétique</term><term>Cellule érythroïde</term><term>Gène</term><term>Globine</term><term>Ankyrine</term><term>Souris</term><term>Animal</term><term>Homme</term><term>Antigène CD34</term></keywords><keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Homme</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Use of oncoretroviral vectors in gene therapy for hemoglobinopathies has been impeded by low titer vectors, genetic instability, and poor expression. Fifteen self-inactivating (SIN) lentiviral vectors using 4 erythroid promoters in combination with 4 erythroid enhancers with or without the woodchuck hepatitis virus postregulatory element (WPRE) were generated using the enhanced green fluorescent protein as a reporter gene. Vectors with high erythroid-specific expression in cell lines were tested in primary human CD34<sup>+</sup> cells and in vivo in the murine bone marrow (BM) transplantation model. Vectors containing the ankyrin-1 promoter showed high-level expression and stable proviral transmission. Two vectors containing the ankyrin-1 promoter and 2 erythroid enhancers (HS-40 plus GATA-1 or HS-40 plus 5-aminolevulinate synthase intron 8 [18] enhancers) and WPRE expressed at levels higher than the HS2/β-promoter vector in bulk unilineage erythroid cultures and individual erythroid blast-forming units derived from human BM CD34<sup>+</sup> cells. Sca1<sup>+</sup>/lineage<sup>-</sup> Ly5.1 mouse hematopoietic cells, transduced with these 2 ankyrin-1 promoter vectors, were injected into lethally irradiated Ly5.2 recipients. Eleven weeks after transplantation, high-level expression was seen from both vectors in blood (63%-89% of red blood cells) and erythroid cells in BM (70%-86% engraftment), compared with negligible expression in myeloid and lymphoid lineages in blood, BM, spleen, and thymus (0%-4%). The I8/HS-40-containing vector encoding a hybrid human [3/y-globin gene led to 43% to 113% human γ-globin expression/copy of the mouse α-globin gene. Thus, modular use of erythroid-specific enhancers/promoters and WPRE in SIN-lentiviral vectors led to identification of high-titer, stably transmitted vectors with high-level erythroid-specific expression for gene therapy of red cell diseases.</div></front></TEI><affiliations><list><country><li>Australie</li><li>Canada</li><li>France</li><li>Italie</li><li>Taïwan</li><li>États-Unis</li></country><region><li>Aquitaine</li><li>Californie</li><li>Lombardie</li><li>Nouvelle-Aquitaine</li></region><settlement><li>Bordeaux</li><li>Los Angeles</li><li>Milan</li></settlement></list><tree><noCountry><name sortKey="Bauer, Gerhard" sort="Bauer, Gerhard" uniqKey="Bauer G" first="Gerhard" last="Bauer">Gerhard Bauer</name><name sortKey="Ellis, James" sort="Ellis, James" uniqKey="Ellis J" first="James" last="Ellis">James Ellis</name><name sortKey="Gang Jiang" sort="Gang Jiang" uniqKey="Gang Jiang" last="Gang Jiang">GANG JIANG</name><name sortKey="Malik, Punam" sort="Malik, Punam" uniqKey="Malik P" first="Punam" last="Malik">Punam Malik</name><name sortKey="Mavilio, Fulvio" sort="Mavilio, Fulvio" uniqKey="Mavilio F" first="Fulvio" last="Mavilio">Fulvio Mavilio</name><name sortKey="Perelman, Natalya P" sort="Perelman, Natalya P" uniqKey="Perelman N" first="Natalya P." last="Perelman">Natalya P. Perelman</name><name sortKey="Ping Xia" sort="Ping Xia" uniqKey="Ping Xia" last="Ping Xia">PING XIA</name><name sortKey="Shen, Che Kun" sort="Shen, Che Kun" uniqKey="Shen C" first="Che-Kun" last="Shen">Che-Kun Shen</name><name sortKey="Surinya, Katherine H" sort="Surinya, Katherine H" uniqKey="Surinya K" first="Katherine H." last="Surinya">Katherine H. Surinya</name></noCountry><country name="États-Unis"><region name="Californie"><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></region></country><country name="France"><region name="Nouvelle-Aquitaine"><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></region></country><country name="Canada"><noRegion><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></noRegion></country><country name="Australie"><noRegion><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></noRegion></country><country name="Italie"><region name="Lombardie"><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></region><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></country><country name="Taïwan"><noRegion><name sortKey="Moreau Gaudry, Francois" sort="Moreau Gaudry, Francois" uniqKey="Moreau Gaudry F" first="Francois" last="Moreau-Gaudry">Francois Moreau-Gaudry</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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