Un nuevo caso de terapia génica

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

 

Disorders caused by abnormal [beta]-globin, such as

[beta]-thalassaemia, are the most prevalent inherited disorders

worldwide. For treatment, many patients are dependent on blood

transfusions; thus far the only cure has involved matched

transplantation of haematopoietic stem cells. Here it is shown that

lentiviral [beta]-globin gene transfer can be an effective substitute

for regular transfusions in a patient with severe [beta]-thalassaemia

 

http://www.nature.com/nature/journal/v467/n7313/abs/nature09328.html

Nature, septiembre del 2010