Different Serum Enzyme Levels Are Required to Rescue the Various Systemic Features of the Mucopolysaccharidoses-Reference-Cited by-同舟云学术

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Different Serum Enzyme Levels Are Required to Rescue the Various Systemic Features of the Mucopolysaccharidoses

Published:2010-05 Issue:5 Volume:21 Page:555-569
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Cotugno Gabriella¹²,Tessitore Alessandra¹,Capalbo Anita¹²,Annunziata Patrizia¹²,Strisciuglio Caterina¹²,Faella Armida¹,Aurilio Michela³,Di Tommaso Maurizio¹,Russo Fabio¹,Mancini Antonio⁴,De Leonibus Elvira¹,Aloj Luigi³,Auricchio Alberto¹²

Affiliation:

1. Telethon Institute of Genetics and Medicine (TIGEM), 80131 Naples, Italy.

2. Medical Genetics, Department of Pediatrics, Federico II University, 80131 Naples, Italy.

3. Department of Nuclear Medicine, Istituto Nazionale Tumori Fondazione G. Pascale, 80131 Naples, Italy.

4. Centro di Biotecnologie, Antonio Cardarelli Hospital, 80131 Naples, Italy.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2009.189

Reference73 articles.

1. Growth plate pathology in feline mucopolysaccharidosis VI

2. Enzyme replacement therapy in 12 patients with MPS I–H/S with homozygous p.Leu490Pro mutation

3. Gene Therapy of Storage Disorders by Retroviral and Lentiviral Vectors

Cited by 24 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Liver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI;NEJM Evidence;2022-06-28

2. Failures of Endochondral Ossification in the Mucopolysaccharidoses;Current Osteoporosis Reports;2020-10-16

3. Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism;Human Gene Therapy;2019-10-01

4. Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases;Genetics and Molecular Biology;2019

5. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI;Molecular Therapy - Methods & Clinical Development;2017-09

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