Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy Journal Article
| Authors: | Sadelain, M.; Boulad, F.; Galanello, R.; Giardina, P.; Locatelli, F.; Maggio, A.; Rivella, S.; Riviere, I.; Tisdale, J. |
| Article Title: | Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy |
| Keywords: | treatment outcome; busulfan; mortality; neutropenia; review; interferon; drug safety; drug withdrawal; hepatitis c; liver cirrhosis; nonhuman; side effect; drug megadose; follow up; animals; mice; disease association; quality of life; liver toxicity; gene expression; anemia; gastrointestinal symptom; morbidity; gene locus; creatinine blood level; continuous infusion; risk assessment; carcinogenesis; gene transfer; viral gene delivery system; alanine aminotransferase blood level; drug hypersensitivity; rash; hla matching; diabetes mellitus; gene therapy; lentivirus vector; beta thalassemia; globin gene; graft versus host reaction; beta-thalassemia; allogeneic hematopoietic stem cell transplantation; osteoporosis; hypogonadism; hepatitis c virus; drug treatment failure; erythrocyte transfusion; genetic screening; bacterial infection; anaphylaxis; disease exacerbation; ribavirin; infection risk; deferoxamine; autologous hematopoietic stem cell transplantation; arthropathy; cardiomyopathy; globins; beta globin; osteopenia; deferasirox; iron overload; agranulocytosis; phlebotomy; cell based gene therapy; deferiprone; iron blood level |
| Journal Title: | Human Gene Therapy |
| Volume: | 18 |
| Issue: | 1 |
| ISSN: | 1043-0342 |
| Publisher: | Mary Ann Liebert, Inc |
| Date Published: | 2007-01-01 |
| Start Page: | 1 |
| End Page: | 9 |
| Language: | English |
| DOI: | 10.1089/hum.2006.151 |
| PUBMED: | 17173507 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | --- - "Cited By (since 1996): 22" - "Export Date: 17 November 2011" - "CODEN: HGTHE" - "Source: Scopus" |
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