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Sanfilippo disease Supported projects Intracerebral Gene Therapy

Intracerebral gene therapy

(Programme organised by Lysogene, the Téléthon France and Prof. Marc Tardieu, Hôpital Necker-Enfants Malades and Hôpital Kremlin Bicêtre, Paris)

The start of the first clinical trial of phase I/II gene therapy for children suffering from MPS IIIA was a marked event for 2011 in Europe. The principle of gene therapy consists of introducing a corrector gene into the brain of sick children which should lead to the production of the missing enzyme in the tissue of the central nervous system. The SAF-301 clinical trial conducted by Prof. Tardieu on four children affected by this form of Sanfilippo disease aimed to evaluate the tolerance to the treatment proposed during the observation period. If the results prove conclusive, a clinical phase III trial will then be extended to a larger number of children in 2013.