Intracerebral Gene Therapy in Four Children with Sanfilippo B Syndrome: 5.5-Year Follow-Up Results

Author:

Deiva Kumaran1ORCID,Ausseil Jérôme23,de Bournonville Stéphanie1,Zérah Michel456,Husson Béatrice7,Gougeon Marie-Lise8,Poirier-Beaudouin Béatrice8,Zafeiriou Dimitrios9,Parenti Giancarlo1011,Heard Jean-Michel12,Tardieu Marc1

Affiliation:

1. Pediatric Neurology Department, Université Paris-Saclay, Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris-Saclay, Site Bicêtre, Le Kremlin-Bicêtre, France.

2. Service de Biochimie, Institut Fédératif de Biologie, Centre Hospitalier Universitaire de Toulouse, Toulouse, France.

3. Inserm U1043 Centre de Physiopathologie de Toulouse-Purpan Université Toulouse III Paul Sabatier, Toulouse, France.

4. Pediatric Neurosurgery Department, Assistance Publique-Hôpitaux de Paris, Hôpital Necker, Paris, France.

5. Institut Imagine, Université René Descartes, Paris, France.

6. NeuroGenCell, Institut du Cerveau et de la Moelle, Paris, France.

7. Pediatric Radiology Department, Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris-Saclay, Le Kremlin-Bicêtre, France.

8. Innate Immunity and Viruses Unit, Global Health Department, Institut Pasteur, Paris, France.

9. Department of Pediatrics, Aristotle University, Thessaloniki, Greece.

10. Department of Translational Medical Sciences, Frederico II University, Naples, Italy.

11. Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.

12. Department of Neuroscience, Biotherapy and Neurodegenerative Diseases Unit, Institut Pasteur, INSERM U1115, Paris, France.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

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