Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene
| Autor: | Yves Beuzard, Olivier Negre, Marina Cavazzana, Philippe Leboulch, Anne-Virginie Eggimann, Philippe Bourget, Emmanuel Payen, Jean-Antoine Ribeil, Salima Hacein-Bey, Suparerk Borwornpinyo, Suradej Hongeng |
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| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
Transplantation Conditioning medicine.medical_treatment Genetic enhancement Transgene Genetic Vectors Gene Expression Reviews Anemia Sickle Cell beta-Globins Hematopoietic stem cell transplantation 03 medical and health sciences hemic and lymphatic diseases Gene expression Genetics medicine Humans Transgenes Molecular Biology Gene Clinical Trials as Topic business.industry Lentivirus beta-Thalassemia Gene Transfer Techniques Hematopoietic Stem Cell Transplantation Beta thalassemia Genetic Therapy Hematopoietic Stem Cells medicine.disease 030104 developmental biology Cancer research Molecular Medicine Biomarker (medicine) Patient Safety Stem cell business |
| Zdroj: | Human Gene Therapy |
| ISSN: | 1557-7422 1043-0342 |
| Popis: | β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-globin gene derivative (β(AT87Q)-globin) to hematopoietic stem cells, driven by cis-regulatory elements that confer high, erythroid-specific expression, has been evaluated in human clinical trials over the past 8 years. β(AT87Q)-globin is used both as a strong inhibitor of HbS polymerization and as a biomarker. While long-term studies are underway in multiple centers in Europe and in the United States, proof-of-principle of efficacy and safety has already been obtained in multiple patients with β-thalassemia and sickle cell disease. |
| Databáze: | OpenAIRE |
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