التفاصيل البيبلوغرافية
| العنوان: |
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia |
| المؤلفون: |
Marina Cavazzana-Calvo, Emmanuel Payen, Olivier Negre, Gary Wang, Kathleen Hehir, Floriane Fusil, Julian Down, Maria Denaro, Troy Brady, Karen Westerman, Resy Cavallesco, Beatrix Gillet-Leg |
| المصدر: |
Nature, Nature. 467(7313):318-322 |
| سنة النشر: |
2010 |
| الوصف: |
Gene therapy success Blood disorders caused by abnormal β-globin — β-thalassaemia and sickle cell disease — are the most prevalent inherited disorders worldwide, with patients often remaining dependent on blood transfusions throughout their lives. So a report of the successful use of gene therapy in a case of severe β-thalassaemia — using a lentiviral vector expressing the β-globin gene — is an eagerly awaited event. More than two years after gene transfer, the adult male patient has been transfusion-independent for 21 months. The therapeutic benefit seems to result from a dominant, myeloid-biased cell clone that may remain benign, although it could yet develop into leukaemia — a reminder that gene therapy is still at an early stage. |
| نوع الوثيقة: |
redif-article |
| اللغة: |
English |
| DOI: |
10.1038/nature09328 |
| الإتاحة: |
https://ideas.repec.org/a/nat/nature/v467y2010i7313d10.1038_nature09328.html |
| رقم الأكسشن: |
edsrep.a.nat.nature.v467y2010i7313d10.1038.nature09328 |
| قاعدة البيانات: |
RePEc |
