NEJM – Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
This is a breakthrough article in the field of Neurology!
Cerebral adrenal leukodystrophy is an X-linked disease with a single gene mutation. I carries a dismal prognosis for the majority of patients, who can not find a suitable bone marrow donor for ALLOGENEIC stem cell transplant.
This paper show results of AUTOLOGOUS stem cell transplant after transduction of the CD34+ cells with lentiviruses that fixed the pathological mutation.
Check results of this cutting-edge research at NEJM: