Intracerebral Gene Therapy in Four Children with Sanfilippo B Syndrome: 5.5-Year Follow-Up Results-Reference-Cited by-同舟云学术

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

Published:2021-10-01 Issue:19-20 Volume:32 Page:1251-1259
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Deiva Kumaran¹^ORCID,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

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2021.135

Reference19 articles.

1. Improved Behavior and Neuropathology in the Mouse Model of Sanfilippo Type IIIB Disease after Adeno-Associated Virus-Mediated Gene Transfer in the Striatum

2. A model of mucopolysaccharidosis IIIB (Sanfilippo syndrome type IIIB): N -acetyl-α-D-glucosaminidase deficiency in Schipperke dogs

3. Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

4. Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome

5. Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes

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