Successful Peripheral T-Lymphocyte–Directed Gene Transfer for a Patient With Severe Combined Immune Deficiency Caused by Adenosine Deaminase Deficiency
| Autor: | Kotani H, Masafumi Yamada, Ichiro Kobayashi, Shuzo Matsumoto, McGarrity Gj, Makoto Ohtsu, Tadashi Ariga, Motohiko Okano, M. Onodera, Atsushi Tame, Hirofumi Furuta, Nobuaki Kawamura, Blaese Rm, Yukio Sakiyama |
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| Rok vydání: | 1998 |
| Předmět: |
Male
Adenosine Deaminase Recombinant Fusion Proteins T-Lymphocytes Genetic enhancement Genetic Vectors Immunology Transfection Transplantation Autologous Biochemistry Viral vector Adenosine deaminase Immune system Immunopathology medicine Humans Hypersensitivity Delayed Lymphocyte Count Cells Cultured Skin Tests Immunity Cellular biology business.industry Genetic transfer Immunoglobulins Intravenous Genetic Therapy Cell Biology Hematology T lymphocyte medicine.disease Combined Modality Therapy Adenosine deaminase deficiency Hemagglutinins Retroviridae Child Preschool Immunoglobulin G Antibody Formation biology.protein Severe Combined Immunodeficiency business |
| Zdroj: | Blood. 91:30-36 |
| ISSN: | 1528-0020 0006-4971 |
| DOI: | 10.1182/blood.v91.1.30 |
| Popis: | Ten patients with adenosine deaminase deficiency (ADA−) have been enrolled in gene therapy clinical trials since the first patient was treated in September 1990. We describe a Japanese ADA− severe combined immune deficiency (SCID) patient who has received periodic infusions of genetically modified autologous T lymphocytes transduced with the human ADA cDNA containing retroviral vector LASN. The percentage of peripheral blood lymphocytes carrying the transduced ADA gene has remained stable at 10% to 20% during the 12 months since the fourth infusion. ADA enzyme activity in the patient's circulating T cells, which was only marginally detected before gene transfer, increased to levels comparable to those of a heterozygous carrier individual and was associated with increased T-lymphocyte counts and improvement of the patient's immune function. The results obtained in this trial are in agreement with previously published observations and support the usefulness of T lymphocyte-directed gene transfer in the treatment of ADA−SCID. |
| Databáze: | OpenAIRE |
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