Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency
Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UKAbstract: The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (S...
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| Format: | Article |
| Language: | English |
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Dove Medical Press
2009-12-01
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| Series: | Stem Cells and Cloning: Advances and Applications |
| Online Access: | http://www.dovepress.com/development-of-gene-therapy-potential-in-severe-combined-immunodeficie-a3855 |
| _version_ | 1818405425917198336 |
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| author | Claudia A Montiel-Equihua Adrian J Thrasher H Bobby Gaspar |
| author_facet | Claudia A Montiel-Equihua Adrian J Thrasher H Bobby Gaspar |
| author_sort | Claudia A Montiel-Equihua |
| collection | DOAJ |
| description | Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UKAbstract: The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (SCID) and especially adenosine deaminase (ADA)-deficient SCID. Here we discuss the developments achieved in over two decades of clinical and laboratory research that led to the establishment of a protocol for the autologous transplant of retroviral vector-mediated gene-modified hematopoietic stem cells, which has proved to be both successful and, to date, safe. Patients in trials in three different countries have shown long-term immunological and metabolic correction. Nevertheless, improvements to the safety profile of viral vectors are underway and will undoubtedly reinforce the position of stem cell gene therapy as a treatment option for ADA-SCID.Keywords: adenosine deaminase, severe combined immunodeficiency, gene therapy, hematopoietic stem cell, retrovirus, clinical trial |
| first_indexed | 2024-12-14T08:55:51Z |
| format | Article |
| id | doaj.art-936454d41ea942afac64b7d8188fefc7 |
| institution | Directory Open Access Journal |
| issn | 1178-6957 |
| language | English |
| last_indexed | 2024-12-14T08:55:51Z |
| publishDate | 2009-12-01 |
| publisher | Dove Medical Press |
| record_format | Article |
| series | Stem Cells and Cloning: Advances and Applications |
| spelling | doaj.art-936454d41ea942afac64b7d8188fefc72022-12-21T23:08:56ZengDove Medical PressStem Cells and Cloning: Advances and Applications1178-69572009-12-012010default112Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiencyClaudia A Montiel-EquihuaAdrian J ThrasherH Bobby GasparClaudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UKAbstract: The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (SCID) and especially adenosine deaminase (ADA)-deficient SCID. Here we discuss the developments achieved in over two decades of clinical and laboratory research that led to the establishment of a protocol for the autologous transplant of retroviral vector-mediated gene-modified hematopoietic stem cells, which has proved to be both successful and, to date, safe. Patients in trials in three different countries have shown long-term immunological and metabolic correction. Nevertheless, improvements to the safety profile of viral vectors are underway and will undoubtedly reinforce the position of stem cell gene therapy as a treatment option for ADA-SCID.Keywords: adenosine deaminase, severe combined immunodeficiency, gene therapy, hematopoietic stem cell, retrovirus, clinical trialhttp://www.dovepress.com/development-of-gene-therapy-potential-in-severe-combined-immunodeficie-a3855 |
| spellingShingle | Claudia A Montiel-Equihua Adrian J Thrasher H Bobby Gaspar Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency Stem Cells and Cloning: Advances and Applications |
| title | Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency |
| title_full | Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency |
| title_fullStr | Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency |
| title_full_unstemmed | Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency |
| title_short | Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency |
| title_sort | development of gene therapy potential in severe combined immunodeficiency due to adenosine deaminase deficiency |
| url | http://www.dovepress.com/development-of-gene-therapy-potential-in-severe-combined-immunodeficie-a3855 |
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