Gene Therapy with Cord Blood Hematopoietic Stem Cells for Adenosine Deaminase Deficiency: An Update
| Autor: | Crooks Gm, Jan A. Nolta, Donald B. Kohn, Robertson Parkman, Kenneth I. Weinberg |
|---|---|
| Rok vydání: | 2000 |
| Předmět: |
chemistry.chemical_classification
congenital hereditary and neonatal diseases and abnormalities business.industry Genetic enhancement nutritional and metabolic diseases hemic and immune systems Disease medicine.disease Phenotype Adenosine deaminase deficiency enzymes and coenzymes (carbohydrates) Haematopoiesis Enzyme chemistry immune system diseases Cord blood Immunology Medicine Stem cell business |
| Zdroj: | Transplantation in Hematology and Oncology ISBN: 9783642640414 |
| DOI: | 10.1007/978-3-642-59592-9_35 |
| Popis: | Gene therapy for ADA deficiency has been a model disease in which to evaluate hematopoietic stem cells for clinical gene therapy [1]. ADA deficiency has been a candidate disease because 1. ADA is a single polypeptide protein, 2. a clinically normal phenotype is associated with a wide range of enzymatic function (5–500% of normal), and 3. ADA is widely expressed so that tissue specific expression is not required. |
| Databáze: | OpenAIRE |
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