Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
Deficiency of adenosine deaminase (ADA) causes a form of severe combined immunodeficiency disease. This study shows that gene therapy in the form of hematopoietic stem cells transduced with a viral vector containing the human ADA gene can restore immune function in patients with this disease. Defici...
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| Veröffentlicht in: | The New England journal of medicine 2009-01, Vol.360 (5), p.447-458 |
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| creator | Aiuti, Alessandro Cattaneo, Federica Galimberti, Stefania Benninghoff, Ulrike Cassani, Barbara Callegaro, Luciano Scaramuzza, Samantha Andolfi, Grazia Mirolo, Massimiliano Brigida, Immacolata Tabucchi, Antonella Carlucci, Filippo Eibl, Martha Aker, Memet Slavin, Shimon Al-Mousa, Hamoud Al Ghonaium, Abdulaziz Ferster, Alina Duppenthaler, Andrea Notarangelo, Luigi Wintergerst, Uwe Buckley, Rebecca H Bregni, Marco Marktel, Sarah Valsecchi, Maria Grazia Rossi, Paolo Ciceri, Fabio Miniero, Roberto Bordignon, Claudio Roncarolo, Maria-Grazia |
| description | Deficiency of adenosine deaminase (ADA) causes a form of severe combined immunodeficiency disease. This study shows that gene therapy in the form of hematopoietic stem cells transduced with a viral vector containing the human
ADA
gene can restore immune function in patients with this disease.
Deficiency of adenosine deaminase causes a form of severe combined immunodeficiency disease. This study shows that gene therapy can restore immune function in patients with the disease.
Adenosine deaminase (ADA) deficiency is a fatal autosomal recessive form of severe combined immunodeficiency (SCID), of which failure to thrive, impaired immune responses, and recurrent infections are characteristics.
1
,
2
Toxic levels of purine metabolites (adenosine and adenine deoxyribonucleotides) due to the deficiency of ADA can cause hepatic, skeletal, neurologic, and behavioral alterations
1
,
3
,
4
and sensorineural deafness.
5
A hematopoietic stem-cell transplant from an HLA-identical sibling, the treatment of choice, is available for only a minority of patients
6
–
8
; the use of alternative donors is associated with a high risk of death or lack of engraftment.
1
,
6
Administration of polyethylene . . . |
| doi_str_mv | 10.1056/NEJMoa0805817 |
| format | Article |
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ADA
gene can restore immune function in patients with this disease.
Deficiency of adenosine deaminase causes a form of severe combined immunodeficiency disease. This study shows that gene therapy can restore immune function in patients with the disease.
Adenosine deaminase (ADA) deficiency is a fatal autosomal recessive form of severe combined immunodeficiency (SCID), of which failure to thrive, impaired immune responses, and recurrent infections are characteristics.
1
,
2
Toxic levels of purine metabolites (adenosine and adenine deoxyribonucleotides) due to the deficiency of ADA can cause hepatic, skeletal, neurologic, and behavioral alterations
1
,
3
,
4
and sensorineural deafness.
5
A hematopoietic stem-cell transplant from an HLA-identical sibling, the treatment of choice, is available for only a minority of patients
6
–
8
; the use of alternative donors is associated with a high risk of death or lack of engraftment.
1
,
6
Administration of polyethylene . . .</description><identifier>ISSN: 0028-4793</identifier><identifier>EISSN: 1533-4406</identifier><identifier>DOI: 10.1056/NEJMoa0805817</identifier><identifier>PMID: 19179314</identifier><identifier>CODEN: NEJMAG</identifier><language>eng</language><publisher>Waltham, MA: Massachusetts Medical Society</publisher><subject>Adenosine Deaminase - deficiency ; Adenosine Deaminase - genetics ; Antigens, CD34 - genetics ; Biological and medical sciences ; Bone marrow ; Bone Marrow Cells - immunology ; Child, Preschool ; Combined Modality Therapy ; Ethics ; Follow-Up Studies ; Gene therapy ; General aspects ; Genetic Therapy ; Genetic Vectors ; Hematopoietic Stem Cell Transplantation ; Humans ; Immunodeficiencies ; Immunodeficiencies. Immunoglobulinopathies ; Immunopathology ; Infant ; Kinases ; Ligands ; Lymphocyte Count ; Medical sciences ; Metabolites ; Patients ; Retroviridae ; Severe Combined Immunodeficiency - immunology ; Severe Combined Immunodeficiency - therapy ; Transduction, Genetic ; Transplantation Conditioning ; Transplants & implants</subject><ispartof>The New England journal of medicine, 2009-01, Vol.360 (5), p.447-458</ispartof><rights>Copyright © 2009 Massachusetts Medical Society. All rights reserved.</rights><rights>2009 INIST-CNRS</rights><rights>2009 Massachusetts Medical Society</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c548t-ecf80bddcbf9ce96fe2b23019c808f4779081dfd4a25864545aca96e27f6f8ad3</citedby><cites>FETCH-LOGICAL-c548t-ecf80bddcbf9ce96fe2b23019c808f4779081dfd4a25864545aca96e27f6f8ad3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.nejm.org/doi/pdf/10.1056/NEJMoa0805817$$EPDF$$P50$$Gmms$$H</linktopdf><linktohtml>$$Uhttps://www.nejm.org/doi/full/10.1056/NEJMoa0805817$$EHTML$$P50$$Gmms$$H</linktohtml><link.rule.ids>314,776,780,2746,2747,26080,27901,27902,52357,54039</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=21107778$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/19179314$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Aiuti, Alessandro</creatorcontrib><creatorcontrib>Cattaneo, Federica</creatorcontrib><creatorcontrib>Galimberti, Stefania</creatorcontrib><creatorcontrib>Benninghoff, Ulrike</creatorcontrib><creatorcontrib>Cassani, Barbara</creatorcontrib><creatorcontrib>Callegaro, Luciano</creatorcontrib><creatorcontrib>Scaramuzza, Samantha</creatorcontrib><creatorcontrib>Andolfi, Grazia</creatorcontrib><creatorcontrib>Mirolo, Massimiliano</creatorcontrib><creatorcontrib>Brigida, Immacolata</creatorcontrib><creatorcontrib>Tabucchi, Antonella</creatorcontrib><creatorcontrib>Carlucci, Filippo</creatorcontrib><creatorcontrib>Eibl, Martha</creatorcontrib><creatorcontrib>Aker, Memet</creatorcontrib><creatorcontrib>Slavin, Shimon</creatorcontrib><creatorcontrib>Al-Mousa, Hamoud</creatorcontrib><creatorcontrib>Al Ghonaium, Abdulaziz</creatorcontrib><creatorcontrib>Ferster, Alina</creatorcontrib><creatorcontrib>Duppenthaler, Andrea</creatorcontrib><creatorcontrib>Notarangelo, Luigi</creatorcontrib><creatorcontrib>Wintergerst, Uwe</creatorcontrib><creatorcontrib>Buckley, Rebecca H</creatorcontrib><creatorcontrib>Bregni, Marco</creatorcontrib><creatorcontrib>Marktel, Sarah</creatorcontrib><creatorcontrib>Valsecchi, Maria Grazia</creatorcontrib><creatorcontrib>Rossi, Paolo</creatorcontrib><creatorcontrib>Ciceri, Fabio</creatorcontrib><creatorcontrib>Miniero, Roberto</creatorcontrib><creatorcontrib>Bordignon, Claudio</creatorcontrib><creatorcontrib>Roncarolo, Maria-Grazia</creatorcontrib><title>Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency</title><title>The New England journal of medicine</title><addtitle>N Engl J Med</addtitle><description>Deficiency of adenosine deaminase (ADA) causes a form of severe combined immunodeficiency disease. This study shows that gene therapy in the form of hematopoietic stem cells transduced with a viral vector containing the human
ADA
gene can restore immune function in patients with this disease.
Deficiency of adenosine deaminase causes a form of severe combined immunodeficiency disease. This study shows that gene therapy can restore immune function in patients with the disease.
Adenosine deaminase (ADA) deficiency is a fatal autosomal recessive form of severe combined immunodeficiency (SCID), of which failure to thrive, impaired immune responses, and recurrent infections are characteristics.
1
,
2
Toxic levels of purine metabolites (adenosine and adenine deoxyribonucleotides) due to the deficiency of ADA can cause hepatic, skeletal, neurologic, and behavioral alterations
1
,
3
,
4
and sensorineural deafness.
5
A hematopoietic stem-cell transplant from an HLA-identical sibling, the treatment of choice, is available for only a minority of patients
6
–
8
; the use of alternative donors is associated with a high risk of death or lack of engraftment.
1
,
6
Administration of polyethylene . . .</description><subject>Adenosine Deaminase - deficiency</subject><subject>Adenosine Deaminase - genetics</subject><subject>Antigens, CD34 - genetics</subject><subject>Biological and medical sciences</subject><subject>Bone marrow</subject><subject>Bone Marrow Cells - immunology</subject><subject>Child, Preschool</subject><subject>Combined Modality Therapy</subject><subject>Ethics</subject><subject>Follow-Up Studies</subject><subject>Gene therapy</subject><subject>General aspects</subject><subject>Genetic Therapy</subject><subject>Genetic Vectors</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Humans</subject><subject>Immunodeficiencies</subject><subject>Immunodeficiencies. Immunoglobulinopathies</subject><subject>Immunopathology</subject><subject>Infant</subject><subject>Kinases</subject><subject>Ligands</subject><subject>Lymphocyte Count</subject><subject>Medical sciences</subject><subject>Metabolites</subject><subject>Patients</subject><subject>Retroviridae</subject><subject>Severe Combined Immunodeficiency - immunology</subject><subject>Severe Combined Immunodeficiency - therapy</subject><subject>Transduction, Genetic</subject><subject>Transplantation Conditioning</subject><subject>Transplants & implants</subject><issn>0028-4793</issn><issn>1533-4406</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2009</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BEC</sourceid><sourceid>BENPR</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNp10EFLwzAUB_AgipvTo1cpgt6qSZukyXFscypTL_NcsvQFO5ZkJuth396OVYeCubzw-PHe44_QJcF3BDN-_zp5fvEKC8wEKY5Qn7A8TynF_Bj1Mc5ESguZ99BZjEvcPkLlKeoRSdouoX00nYKDZP4BQa23ifEhebK2cb4CU-sanN4m4waSjU-GFTgf61aPQdnaqbj7fatzdGLUKsJFVwfo_WEyHz2ms7fp02g4SzWjYpOCNgIvqkovjNQguYFskeWYSC2wMLQoJBakMhVVGROcMsqUVpJDVhhuhKryAbrdz10H_9lA3JS2jhpWK-XAN7HkXHCGKW_h9R-49E1w7W1lluWS5FSyFqV7pIOPMYAp16G2KmxLgstdvOWveFt_1Q1tFhaqg-7ybMFNB1TUamWCcrqOPy4jBBdFIQ7O2lg6WNp_Fn4B-faNIg</recordid><startdate>20090129</startdate><enddate>20090129</enddate><creator>Aiuti, Alessandro</creator><creator>Cattaneo, Federica</creator><creator>Galimberti, Stefania</creator><creator>Benninghoff, Ulrike</creator><creator>Cassani, Barbara</creator><creator>Callegaro, Luciano</creator><creator>Scaramuzza, Samantha</creator><creator>Andolfi, Grazia</creator><creator>Mirolo, Massimiliano</creator><creator>Brigida, Immacolata</creator><creator>Tabucchi, Antonella</creator><creator>Carlucci, Filippo</creator><creator>Eibl, Martha</creator><creator>Aker, Memet</creator><creator>Slavin, Shimon</creator><creator>Al-Mousa, Hamoud</creator><creator>Al Ghonaium, Abdulaziz</creator><creator>Ferster, Alina</creator><creator>Duppenthaler, Andrea</creator><creator>Notarangelo, Luigi</creator><creator>Wintergerst, Uwe</creator><creator>Buckley, Rebecca H</creator><creator>Bregni, Marco</creator><creator>Marktel, Sarah</creator><creator>Valsecchi, Maria Grazia</creator><creator>Rossi, Paolo</creator><creator>Ciceri, Fabio</creator><creator>Miniero, Roberto</creator><creator>Bordignon, Claudio</creator><creator>Roncarolo, Maria-Grazia</creator><general>Massachusetts Medical Society</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>0TZ</scope><scope>7RV</scope><scope>7X7</scope><scope>7XB</scope><scope>8AO</scope><scope>8C1</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BEC</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>HCIFZ</scope><scope>K0Y</scope><scope>LK8</scope><scope>M0R</scope><scope>M0T</scope><scope>M1P</scope><scope>M2M</scope><scope>M2O</scope><scope>M2P</scope><scope>M7P</scope><scope>MBDVC</scope><scope>NAPCQ</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>PSYQQ</scope><scope>Q9U</scope><scope>7X8</scope></search><sort><creationdate>20090129</creationdate><title>Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency</title><author>Aiuti, Alessandro ; Cattaneo, Federica ; Galimberti, Stefania ; Benninghoff, Ulrike ; Cassani, Barbara ; Callegaro, Luciano ; Scaramuzza, Samantha ; Andolfi, Grazia ; Mirolo, Massimiliano ; Brigida, Immacolata ; Tabucchi, Antonella ; Carlucci, Filippo ; Eibl, Martha ; Aker, Memet ; Slavin, Shimon ; Al-Mousa, Hamoud ; Al Ghonaium, Abdulaziz ; Ferster, Alina ; Duppenthaler, Andrea ; Notarangelo, Luigi ; Wintergerst, Uwe ; Buckley, Rebecca H ; Bregni, Marco ; Marktel, Sarah ; Valsecchi, Maria Grazia ; Rossi, Paolo ; Ciceri, Fabio ; Miniero, Roberto ; Bordignon, Claudio ; Roncarolo, Maria-Grazia</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c548t-ecf80bddcbf9ce96fe2b23019c808f4779081dfd4a25864545aca96e27f6f8ad3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2009</creationdate><topic>Adenosine Deaminase - deficiency</topic><topic>Adenosine Deaminase - genetics</topic><topic>Antigens, CD34 - genetics</topic><topic>Biological and medical sciences</topic><topic>Bone marrow</topic><topic>Bone Marrow Cells - immunology</topic><topic>Child, Preschool</topic><topic>Combined Modality Therapy</topic><topic>Ethics</topic><topic>Follow-Up Studies</topic><topic>Gene therapy</topic><topic>General aspects</topic><topic>Genetic Therapy</topic><topic>Genetic Vectors</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Humans</topic><topic>Immunodeficiencies</topic><topic>Immunodeficiencies. Immunoglobulinopathies</topic><topic>Immunopathology</topic><topic>Infant</topic><topic>Kinases</topic><topic>Ligands</topic><topic>Lymphocyte Count</topic><topic>Medical sciences</topic><topic>Metabolites</topic><topic>Patients</topic><topic>Retroviridae</topic><topic>Severe Combined Immunodeficiency - immunology</topic><topic>Severe Combined Immunodeficiency - therapy</topic><topic>Transduction, Genetic</topic><topic>Transplantation Conditioning</topic><topic>Transplants & implants</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Aiuti, Alessandro</creatorcontrib><creatorcontrib>Cattaneo, Federica</creatorcontrib><creatorcontrib>Galimberti, Stefania</creatorcontrib><creatorcontrib>Benninghoff, Ulrike</creatorcontrib><creatorcontrib>Cassani, Barbara</creatorcontrib><creatorcontrib>Callegaro, Luciano</creatorcontrib><creatorcontrib>Scaramuzza, Samantha</creatorcontrib><creatorcontrib>Andolfi, Grazia</creatorcontrib><creatorcontrib>Mirolo, Massimiliano</creatorcontrib><creatorcontrib>Brigida, Immacolata</creatorcontrib><creatorcontrib>Tabucchi, Antonella</creatorcontrib><creatorcontrib>Carlucci, Filippo</creatorcontrib><creatorcontrib>Eibl, Martha</creatorcontrib><creatorcontrib>Aker, Memet</creatorcontrib><creatorcontrib>Slavin, Shimon</creatorcontrib><creatorcontrib>Al-Mousa, Hamoud</creatorcontrib><creatorcontrib>Al Ghonaium, Abdulaziz</creatorcontrib><creatorcontrib>Ferster, Alina</creatorcontrib><creatorcontrib>Duppenthaler, Andrea</creatorcontrib><creatorcontrib>Notarangelo, Luigi</creatorcontrib><creatorcontrib>Wintergerst, Uwe</creatorcontrib><creatorcontrib>Buckley, Rebecca H</creatorcontrib><creatorcontrib>Bregni, Marco</creatorcontrib><creatorcontrib>Marktel, Sarah</creatorcontrib><creatorcontrib>Valsecchi, Maria Grazia</creatorcontrib><creatorcontrib>Rossi, Paolo</creatorcontrib><creatorcontrib>Ciceri, Fabio</creatorcontrib><creatorcontrib>Miniero, Roberto</creatorcontrib><creatorcontrib>Bordignon, Claudio</creatorcontrib><creatorcontrib>Roncarolo, Maria-Grazia</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>Pharma and Biotech Premium PRO</collection><collection>Nursing & Allied Health Database</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>ProQuest Pharma Collection</collection><collection>Public Health Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>British Nursing Database</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>eLibrary</collection><collection>ProQuest Central</collection><collection>Natural Science Collection</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>SciTech Premium Collection</collection><collection>New England Journal of Medicine</collection><collection>ProQuest Biological Science Collection</collection><collection>Consumer Health Database</collection><collection>Healthcare Administration Database</collection><collection>Medical Database</collection><collection>ProQuest Psychology</collection><collection>Research Library</collection><collection>Science Database</collection><collection>Biological Science Database</collection><collection>Research Library (Corporate)</collection><collection>Nursing & Allied Health Premium</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>ProQuest One Psychology</collection><collection>ProQuest Central Basic</collection><collection>MEDLINE - Academic</collection><jtitle>The New England journal of medicine</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Aiuti, Alessandro</au><au>Cattaneo, Federica</au><au>Galimberti, Stefania</au><au>Benninghoff, Ulrike</au><au>Cassani, Barbara</au><au>Callegaro, Luciano</au><au>Scaramuzza, Samantha</au><au>Andolfi, Grazia</au><au>Mirolo, Massimiliano</au><au>Brigida, Immacolata</au><au>Tabucchi, Antonella</au><au>Carlucci, Filippo</au><au>Eibl, Martha</au><au>Aker, Memet</au><au>Slavin, Shimon</au><au>Al-Mousa, Hamoud</au><au>Al Ghonaium, Abdulaziz</au><au>Ferster, Alina</au><au>Duppenthaler, Andrea</au><au>Notarangelo, Luigi</au><au>Wintergerst, Uwe</au><au>Buckley, Rebecca H</au><au>Bregni, Marco</au><au>Marktel, Sarah</au><au>Valsecchi, Maria Grazia</au><au>Rossi, Paolo</au><au>Ciceri, Fabio</au><au>Miniero, Roberto</au><au>Bordignon, Claudio</au><au>Roncarolo, Maria-Grazia</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency</atitle><jtitle>The New England journal of medicine</jtitle><addtitle>N Engl J Med</addtitle><date>2009-01-29</date><risdate>2009</risdate><volume>360</volume><issue>5</issue><spage>447</spage><epage>458</epage><pages>447-458</pages><issn>0028-4793</issn><eissn>1533-4406</eissn><coden>NEJMAG</coden><abstract>Deficiency of adenosine deaminase (ADA) causes a form of severe combined immunodeficiency disease. This study shows that gene therapy in the form of hematopoietic stem cells transduced with a viral vector containing the human
ADA
gene can restore immune function in patients with this disease.
Deficiency of adenosine deaminase causes a form of severe combined immunodeficiency disease. This study shows that gene therapy can restore immune function in patients with the disease.
Adenosine deaminase (ADA) deficiency is a fatal autosomal recessive form of severe combined immunodeficiency (SCID), of which failure to thrive, impaired immune responses, and recurrent infections are characteristics.
1
,
2
Toxic levels of purine metabolites (adenosine and adenine deoxyribonucleotides) due to the deficiency of ADA can cause hepatic, skeletal, neurologic, and behavioral alterations
1
,
3
,
4
and sensorineural deafness.
5
A hematopoietic stem-cell transplant from an HLA-identical sibling, the treatment of choice, is available for only a minority of patients
6
–
8
; the use of alternative donors is associated with a high risk of death or lack of engraftment.
1
,
6
Administration of polyethylene . . .</abstract><cop>Waltham, MA</cop><pub>Massachusetts Medical Society</pub><pmid>19179314</pmid><doi>10.1056/NEJMoa0805817</doi><tpages>12</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0028-4793 |
| ispartof | The New England journal of medicine, 2009-01, Vol.360 (5), p.447-458 |
| issn | 0028-4793 1533-4406 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_66865046 |
| source | MEDLINE; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; New England Journal of Medicine |
| subjects | Adenosine Deaminase - deficiency Adenosine Deaminase - genetics Antigens, CD34 - genetics Biological and medical sciences Bone marrow Bone Marrow Cells - immunology Child, Preschool Combined Modality Therapy Ethics Follow-Up Studies Gene therapy General aspects Genetic Therapy Genetic Vectors Hematopoietic Stem Cell Transplantation Humans Immunodeficiencies Immunodeficiencies. Immunoglobulinopathies Immunopathology Infant Kinases Ligands Lymphocyte Count Medical sciences Metabolites Patients Retroviridae Severe Combined Immunodeficiency - immunology Severe Combined Immunodeficiency - therapy Transduction, Genetic Transplantation Conditioning Transplants & implants |
| title | Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency |
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