Blood Cell Biochemistry: Hematopoiesis and Gene Therapy
Since the first concepts of gene therapy were formulated, the hemopoietic system has been considered the most natural first target tissue for genetic manipulation. The reasons for this include the fact that a very large number of inherited disorders (including some of the most common disorders, such...
Gespeichert in:
| Weitere Verfasser: | , |
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| Format: | Elektronisch E-Book |
| Sprache: | English |
| Veröffentlicht: |
Boston, MA
Springer US
1999
|
| Schriftenreihe: | Blood Cell Biochemistry
8 |
| Schlagworte: | |
| Online-Zugang: | UBR01 Volltext |
| Zusammenfassung: | Since the first concepts of gene therapy were formulated, the hemopoietic system has been considered the most natural first target tissue for genetic manipulation. The reasons for this include the fact that a very large number of inherited disorders (including some of the most common disorders, such as the hemoglobinopathies) are disorders of the hemopoietic system, and the large amount of experience in hematopoietic transplantation biology. The consequence of this resulted in the first clinical trial of gene therapy in 1989, where two children suffering from severe combined immune deficiency (ADA-SCID) were transplanted with T-cells express ing adenosine deaminase (the defective enzyme in patients with this disorder). The partial success of this treatment was perhaps responsible for undue optimism among those proposing other gene therapy treatments within the hematopoietic system, and it has since become clear that there are a number of technical and biological difficulties to overcome before hematopoietic gene therapy becomes a mainstream therapeutic strategy. The chapters in this book evaluate the need for gene therapy in the hematopoietic system, discuss how efficient gene transfer and expression can be achieved in the target cells, highlight areas of difficulty to be addressed, and examine a number of potential applications of the gene therapy approach. The book begins with a chapter by Testa and colleagues, discussing the various sources of hematopoietic cells for both transplantation and gene therapy |
| Beschreibung: | 1 Online-Ressource (XVI, 380 p) |
| ISBN: | 9781461548898 |
| DOI: | 10.1007/978-1-4615-4889-8 |
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| 520 | |a Since the first concepts of gene therapy were formulated, the hemopoietic system has been considered the most natural first target tissue for genetic manipulation. The reasons for this include the fact that a very large number of inherited disorders (including some of the most common disorders, such as the hemoglobinopathies) are disorders of the hemopoietic system, and the large amount of experience in hematopoietic transplantation biology. The consequence of this resulted in the first clinical trial of gene therapy in 1989, where two children suffering from severe combined immune deficiency (ADA-SCID) were transplanted with T-cells express ing adenosine deaminase (the defective enzyme in patients with this disorder). The partial success of this treatment was perhaps responsible for undue optimism among those proposing other gene therapy treatments within the hematopoietic system, and it has since become clear that there are a number of technical and biological difficulties to overcome before hematopoietic gene therapy becomes a mainstream therapeutic strategy. The chapters in this book evaluate the need for gene therapy in the hematopoietic system, discuss how efficient gene transfer and expression can be achieved in the target cells, highlight areas of difficulty to be addressed, and examine a number of potential applications of the gene therapy approach. The book begins with a chapter by Testa and colleagues, discussing the various sources of hematopoietic cells for both transplantation and gene therapy | ||
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| spelling | Blood Cell Biochemistry Hematopoiesis and Gene Therapy edited by Leslie J. Fairbairn, Nydia G. Testa Boston, MA Springer US 1999 1 Online-Ressource (XVI, 380 p) txt rdacontent c rdamedia cr rdacarrier Blood Cell Biochemistry 8 Since the first concepts of gene therapy were formulated, the hemopoietic system has been considered the most natural first target tissue for genetic manipulation. The reasons for this include the fact that a very large number of inherited disorders (including some of the most common disorders, such as the hemoglobinopathies) are disorders of the hemopoietic system, and the large amount of experience in hematopoietic transplantation biology. The consequence of this resulted in the first clinical trial of gene therapy in 1989, where two children suffering from severe combined immune deficiency (ADA-SCID) were transplanted with T-cells express ing adenosine deaminase (the defective enzyme in patients with this disorder). The partial success of this treatment was perhaps responsible for undue optimism among those proposing other gene therapy treatments within the hematopoietic system, and it has since become clear that there are a number of technical and biological difficulties to overcome before hematopoietic gene therapy becomes a mainstream therapeutic strategy. The chapters in this book evaluate the need for gene therapy in the hematopoietic system, discuss how efficient gene transfer and expression can be achieved in the target cells, highlight areas of difficulty to be addressed, and examine a number of potential applications of the gene therapy approach. The book begins with a chapter by Testa and colleagues, discussing the various sources of hematopoietic cells for both transplantation and gene therapy Internal Medicine Oncology Hematology Human Genetics Internal medicine Oncology Human genetics Gentherapie (DE-588)4296957-8 gnd rswk-swf Blutbildendes Gewebe (DE-588)4239650-5 gnd rswk-swf Blutbildendes Gewebe (DE-588)4239650-5 s Gentherapie (DE-588)4296957-8 s DE-604 Fairbairn, Leslie J. edt Testa, Nydia G. edt Erscheint auch als Druck-Ausgabe 9781461372189 Erscheint auch als Druck-Ausgabe 9780306459627 Erscheint auch als Druck-Ausgabe 9781461548904 https://doi.org/10.1007/978-1-4615-4889-8 Verlag URL des Erstveröffentlichers Volltext |
| spellingShingle | Blood Cell Biochemistry Hematopoiesis and Gene Therapy Internal Medicine Oncology Hematology Human Genetics Internal medicine Oncology Human genetics Gentherapie (DE-588)4296957-8 gnd Blutbildendes Gewebe (DE-588)4239650-5 gnd |
| subject_GND | (DE-588)4296957-8 (DE-588)4239650-5 |
| title | Blood Cell Biochemistry Hematopoiesis and Gene Therapy |
| title_auth | Blood Cell Biochemistry Hematopoiesis and Gene Therapy |
| title_exact_search | Blood Cell Biochemistry Hematopoiesis and Gene Therapy |
| title_full | Blood Cell Biochemistry Hematopoiesis and Gene Therapy edited by Leslie J. Fairbairn, Nydia G. Testa |
| title_fullStr | Blood Cell Biochemistry Hematopoiesis and Gene Therapy edited by Leslie J. Fairbairn, Nydia G. Testa |
| title_full_unstemmed | Blood Cell Biochemistry Hematopoiesis and Gene Therapy edited by Leslie J. Fairbairn, Nydia G. Testa |
| title_short | Blood Cell Biochemistry |
| title_sort | blood cell biochemistry hematopoiesis and gene therapy |
| title_sub | Hematopoiesis and Gene Therapy |
| topic | Internal Medicine Oncology Hematology Human Genetics Internal medicine Oncology Human genetics Gentherapie (DE-588)4296957-8 gnd Blutbildendes Gewebe (DE-588)4239650-5 gnd |
| topic_facet | Internal Medicine Oncology Hematology Human Genetics Internal medicine Oncology Human genetics Gentherapie Blutbildendes Gewebe |
| url | https://doi.org/10.1007/978-1-4615-4889-8 |
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