Gene therapy saves two children from adrenoleukodystrophy.

Gene therapy saves two children from adrenoleukodystrophy

Two children with adrenoleukodystrophy have been saved from certain death thanks to gene therapy ... and an inactivated derivative of the AIDS virus!

Adrenoleukodystrophy (ALD) is a rare but deadly genetic disease. It leads to the progressive destruction of the central nervous system (spinal cord and brain), leading to loss of cognitive and motor functions and death. Bone marrow transplantation is not enough: donors are too rare and the risk of complications is high.

A new treatment has just been tested on two children, at the Saint-Vincent-de-Paul hospital in Paris, by French research teams associating Inserm, the Assistance Publique Hôpitaux de Paris and the Université Paris- Descartes. Stem cells from the bone marrow of small patients were removed, modified in the laboratory and then reinjected into the patients.

A vector - a sort of vehicle carrying genetic information - derived from the AIDS virus was associated with it. The AIDS virus is indeed the only one able to penetrate into the nucleus of cells that do not divide: stem cells and neurons. These, reached by the ALD, thus received the therapeutic gene created by the researchers. No side effects have been observed so far.

This world first offers treatment perspectives for all leukodystrophies, as well as for other more frequent diseases (hemophilia, Parkinson ...). The European Association Against Leukodystrophies (ELA) is pleased with this result and the resulting perspectives. It intends to continue to support research, and thus extend testing to the whole of Europe.

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