July 28, 2017
Bulletin interne de l'Institut Pasteur
Gene therapy: first results in children with Sanfilippo B syndrome
On July 13, 2017, the journal Lancet Neurology published the results of a gene therapy trial conducted on four children with Sanfilippo B syndrome (also known as MPS IIIB). This trial marked the culmination of two decades of partnership and financial support from AFM-Téléthon, with the cooperation of the charity "Vaincre les Maladies Lysosomales" (VML). After monitoring the children for 30 months, Dr. Jean-Michel Heard, from the Institut Pasteur and Inserm, and Professors Marc Tardieu and Michel Zérah, from the Paris public hospital network (AP-HP) and Paris-Sud and Paris Descartes Universities, observed a high degree of tolerance to treatment and neurocognitive benefits for patients.