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A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

by Thrasher, Adrian J , Moshous, Despina , Rivat, Christine , Harris, Chad E , Hopkins, Gregory , Cros, Guilhem , Male, Frances , Xu-Bayford, Jinhua , Notarangelo, Luigi D , Fischer, Alain , De Oliveira, Satiro , Ritz, Jerome , Malani, Nirav , Guo, Dongjing , Gaspar, H. Bobby , Marinovic, M. Angélica , Oleastro, Matías , Caccavelli, Laure , Williams, David A , Blanche, Stephane , Shaw, Kit L , Baum, Christopher , Six, Emmanuelle , Tsytsykova, Alla , Schambach, Axel , Filipovich, Alexandra H , Malik, Punam , Neven, Benedicte , Bushman, Frederic D , Cavazzana, Marina , Armant, Myriam , McNicol, Anne-Marie , Blondeau, Johanna , Berry, Charles C , de Boer, Helen , Lim, Annick , Pai, Sung-Yun , Lehmann, Leslie E , Sherman, Eric A , Fernández, Karen S , Hacein-Bey-Abina, Salima , Touzot, Fabien , Buckland, Karen F , van der Loo, Johannes C.M , Picard, Capucine , Silberstein, Leslie E , London, Wendy B , Bleesing, Jack , Kohn, Donald B

in Animals / Antigens, CD34 / Biological and medical sciences / Blood & organ donations / Bone marrow / Cancer / CD3 antigen / CD34 antigen / Children / Children & youth / Clinical trials / Complementary DNA / Deoxyribonucleic acid / DNA / DNA, Complementary - therapeutic use / Families & family life / Gammaretrovirus - genetics / Gene Expression / Gene Silencing / Gene therapy / General aspects / Genetic Therapy - adverse effects / Genetic Vectors / Hematology / Hospitals / Humans / Immunology / Infant / Infections / Insertion / Interleukin 2 / Interleukin Receptor Common gamma Subunit - genetics / Laboratories / Leukemia / Leukemogenesis / Lymphocytes / Lymphocytes T / Male / Medical sciences / Mice / Mutagenesis / Mutation / Pediatrics / Peripheral blood / Preventive medicine / Proto-oncogenes / Severe combined immunodeficiency / T-Lymphocytes - immunology / Transduction, Genetic / Transgenes - physiology / X-Linked Combined Immunodeficiency Diseases - genetics / X-Linked Combined Immunodeficiency Diseases - immunology / X-Linked Combined Immunodeficiency Diseases - therapy

2014

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A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

2014

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A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

2014

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Journal Article

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Thrasher, Adrian J,

Moshous, Despina,

Rivat, Christine,

Harris, Chad E,

Hopkins, Gregory,

Cros, Guilhem,

Male, Frances,

Xu-Bayford, Jinhua,

Notarangelo, Luigi D,

Fischer, Alain,

De Oliveira, Satiro,

Ritz, Jerome,

Malani, Nirav,

Guo, Dongjing,

Gaspar, H. Bobby,

Marinovic, M. Angélica,

Oleastro, Matías,

Caccavelli, Laure,

Williams, David A,

Blanche, Stephane,

Shaw, Kit L,

Baum, Christopher,

Six, Emmanuelle,

Tsytsykova, Alla,

Schambach, Axel,

Filipovich, Alexandra H,

Malik, Punam,

Neven, Benedicte,

Bushman, Frederic D,

Cavazzana, Marina,

Armant, Myriam,

McNicol, Anne-Marie,

Blondeau, Johanna,

Berry, Charles C,

de Boer, Helen,

Lim, Annick,

Pai, Sung-Yun,

Lehmann, Leslie E,

Sherman, Eric A,

Fernández, Karen S,

Hacein-Bey-Abina, Salima,

Touzot, Fabien,

Buckland, Karen F,

van der Loo, Johannes C.M,

Picard, Capucine,

Silberstein, Leslie E,

London, Wendy B,

Bleesing, Jack,

Kohn, Donald B

2014

Overview

Correction of the genetic lesion leading to X-linked SCID with first-generation retroviral vectors has been associated with a 25% risk of acute leukemia. A self-inactivating retrovirus appears to retain therapeutic efficacy, with no leukemia yet observed. X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the gene encoding the interleukin-2 receptor γ chain ( IL2RG ) that result in a lack of response to common γ-chain (γc)–dependent cytokines, an absence of T-cell and natural killer (NK)–cell development, and impairment of B-cell function. 1 , 2 Death from community-acquired or opportunistic infection usually occurs before 1 year of age unless allogeneic hematopoietic stem-cell transplantation (HSCT) is performed. The immunologic defect in children with SCID-X1 obviates the requirement for a preparative regimen before transplantation. 3 – 6 Standard allogeneic HSCT with matched-sibling donors is associated with an 85 to 90% overall . . .

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Publisher

Massachusetts Medical Society

Subject

Animals

/ Antigens, CD34

/ Biological and medical sciences

/ Blood & organ donations

/ Bone marrow

/ Cancer

/ CD3 antigen