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:

http://www.nejm.org/doi/full/10.1056/NEJMoa1700554



Categories: Neurology, Neuroscience, Pediatric

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