Gene therapy against demyelination

A recent publication in the journal Science reported that gene therapy researchers were able to treat two boys diagnosed with X-Linked Adrenoleukodystrophy (also known as Addison-Schilder Disease), a demyelinating disease that progressively damages the myelin, a fatty neural tissue that insulates many nerves of the central and peripheral nervous systems, eventually destroying it.

Dr. Carier and colleagues were able to correct the aberrant gene in stem cells coming from the subjects themselves using a lentiviral vector derived from HIV. Approximately one year after this treatment, the progressive demyelination in the brain stopped and the neurological functions were stabilized. Although more subjects and more follow-up are needed to evaluate the impact of this approach, this study proves that gene therapy can be a successful approach in some diseases.

Original publication: Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrère F, Blanche S, Audit M, Payen E, Leboulch P, l'Homme B, Bougnères P, Von Kalle C, Fischer A, Cavazzana-Calvo M, Aubourg P. Science. 2009 Nov 6;326(5954):818-23.