Liver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI-Reference-Cited by-同舟云学术

Liver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI

Published:2022-06-28 Issue:7 Volume:1 Page:
ISSN:2766-5526
Container-title:NEJM Evidence
language:en
Short-container-title:NEJM Evidence

Author:

Brunetti-Pierri Nicola¹²,Ferla Rita¹²,Ginocchio Virginia Maria¹²,Rossi Alessandro²,Fecarotta Simona³,Romano Roberta²,Parenti Giancarlo¹²,Yildiz Yilmaz⁴,Zancan Stefano⁵,Pecorella Valentina¹,Dell’Anno Margherita¹²,Graziano Mafalda¹,Alliegro Marialuisa¹,Andria Generoso²,Santamaria Francesca²,Brunetti-Pierri Raffaella⁶,Simonelli Francesca⁶,Nigro Vincenzo¹⁷,Vargas Maria⁸,Servillo Giuseppe⁸,Borgia Francesco⁹,Soscia Ernesto¹⁰,Gargaro Marco¹¹,Funghini Silvia¹²,Tedesco Novella¹³¹⁴,Le Brun Pierre Romain¹³¹⁴,Rupar Charles A.¹⁵,Prasad Chitra¹⁵,O’Callaghan Mar¹⁶,Mitchell John J.¹⁷¹⁸,Danos Olivier¹⁹,Marteau Jean-Brice²⁰,Galimberti Stefania²¹,Valsecchi Maria Grazia²¹,Veron Philippe¹³¹⁴,Mingozzi Federico¹³,Fallarino Francesca¹¹,la Marca Giancarlo¹²²²,Sivri H. Serap⁴,Auricchio Alberto¹²³

Affiliation:

1. Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy

2. Department of Translational Medicine, “Federico II” University, Naples, Italy

3. Dipartimento ad Attivitá Integrata Materno Infantile, “Federico II” University, Naples, Italy

4. Division of Pediatric Metabolism, Ihsan Dogramaci Children’s Hospital, Hacettepe University, Ankara, Turkey

5. Fondazione Telethon ETS, Rome, Italy

6. Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Università degli Studi della Campania “Luigi Vanvitelli,” Naples, Italy

7. Department of Precision Medicine, Università degli Studi della Campania “Luigi Vanvitelli,” Naples, Italy

8. Department of Neurosciences, Reproductive and Odontostomatological Sciences, “Federico II” University, Naples, Italy

9. Department of Advanced Biomedical Sciences, Divisions of Cardiology and Cardiothoracic Surgery, “Federico II” University, Naples, Italy

10. Institute of Biostructure and Bioimaging, National Research Council, Napoli, Italy

11. Department of Medicine and Surgery, University of Perugia, Perugia, Italy

12. Newborn Screening, Clinical Chemistry and Pharmacology Laboratory, Meyer Children’s University Hospital, Florence, Italy

13. Genethon, Evry, France

14. Integrare Research Unit UMR_S951 of Université Paris-Saclay, University Evry, INSERM, Evry, France

15. London Health Science Centre, Western University, London, Ontario, Canada

16. Servicio de Neurología, Unidad de Enfermedades Metabólicas, Clínica Rett, Hospital Sant Joan de Déu, Barcelona, Spain

17. Division of Medical Genetics, Montreal Children’s Hospital, McGill University Health Centre, Montreal, Quebec, Canada

18. Division of Pediatric Endocrinology, Montreal Children’s Hospital, McGill University Health Centre, Montreal, Quebec, Canada

19. REGENXBIO Inc, Rockville, MD

20. Genosafe SAS, Evry, France

21. Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy

22. Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy

23. Department of Advanced Biomedical Sciences, “Federico II” University, Naples, Italy

Publisher

Massachusetts Medical Society

Reference33 articles.

1. Mucopolysaccharidosis VI pathophysiology diagnosis and treatment

2. Neufeld E, Muenzer J. The mucopolysaccharidoses. In: Scriver, R, ed. The online metabolic and molecular bases of inherited disease. New York: McGraw Hill, 2010:1-73.

3. Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

4. Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy

5. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance

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