Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Emerging Treatment Strategies-Reference-Cited by-同舟云学术

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Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Emerging Treatment Strategies

Published:2011-06-01 Issue:6 Volume:12 Page:923-930
ISSN:1389-2010
Container-title:Current Pharmaceutical Biotechnology
language:en
Short-container-title:CPB

Author:

de Ruijter J.,J. Valstar M.,A. Wijburg F.

Publisher

Bentham Science Publishers Ltd.

Subject

Pharmaceutical Science,Biotechnology

Cited by 31 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. AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains;Molecular Therapy - Methods & Clinical Development;2020-06

2. In VivoGene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon;Human Gene Therapy;2019-10-01

3. An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA;Human Gene Therapy;2019-09-01

4. Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial;Molecular Genetics and Metabolism;2019-02

5. Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA;International Journal of Molecular Sciences;2017-12-01

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