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在“Elsevier电子期刊”中，命中：483条，耗时：小于0.01 秒

在所有数据库中总计命中：664条

1.Design and rationale of ongoing observational and treatment studies for BMN 250: a novel enzyme replacement therapy for Sanfilippo syndrome type B

作者：Adam Shaywitz ; Stephen Maricich ; Hua Yu ; Steve Kent

刊名：Molecular Genetics and Metabolism

出版年：2017

2.Development of a stem cell gene therapy for Sanfilippo syndrome type B

作者：Don L Clarke ; Valentina Sanguez ; Shih Kan ; Steven Lee ; Michelina Iacovino ; Patricia Dickson

刊名：Molecular Genetics and Metabolism

出版年：2017

3.In Sanfilippo syndrome, heparan sulfate hexasaccharides are the most pathogenic fractions involved in glia activation.

作者：Vincent Puy^a ; Walaa Darwiche^b ; Pierre-Edouard Bodet^c ; Christelle Lony^b ; Cathy Gomila^b ; Sandrine Vitry^d ; Florence Gonnet^c ; Stephanie Trudel^b ; ^e ; Regis Daniel^c ; Jerome Ausseil^b ; ^e

刊名：Molecular Genetics and Metabolism

出版年：2017

4.Design and rational of the natural history study in preparation for the pivotal clinical trial for the treatment of Sanfilippo syndrome type A

作者：Soraya Bekkali^a ; Samantha Parker^a ; Anupam Chakrapani^b ; Roberto Giugliani^c ; Benedicte Heron^d ; Nicole Muschol^e ; Charlotte Roberts^f ; Frits Wijburg^g

刊名：Molecular Genetics and Metabolism

出版年：2017

5.Natural history of siblings with mucopolysaccharidosis type IIIA, Sanfilippo syndrome type A

作者：Alexandra Gossler^a ; Anja Koehn^a ; Ann Meyer^b ; Kai Kossow^a ; Kurt Ullrich^a ; Nicole Muschol^a

刊名：Molecular Genetics and Metabolism

出版年：2017

6.Sanfilippo syndrome type B: a review of patients diagnosed by the MPS Brazil Network

作者：Andressa Federhen^a ; Fabiano Oliveira Poswar^b ; Franciele Barbosa Trapp^c ; Heluí ; sa Castagnino da Rosa^c ; Laysla Pedelhes Silva^c ; Daniele Lima Rocha^c ; Maira Graeff Burin^d ; Regis R Guidobono^d ; Jurema F De Mari^d ; Fernanda Bitencourt^d ; Sandra Leistner-Segal^d ; Ana Carolina Brusius Facchin^d ; Ursula Silveira Matte^b ; Roberto Giugliani^d

刊名：Molecular Genetics and Metabolism

出版年：2017

7.The chitinases expression is related to Simian Immunodeficiency Virus Encephalitis (SIVE) and in HIV encephalitis (HIVE)

作者：C. Sanfilippo^a ; G. Nunnari^d ; A. Calcagno^e ; L. Malaguarnera^b ; K. Blennow^c ; H. Zetterberg^c ; M. Di Rosa^b ; ^{mdirosa@unict.it}

关键词：HIV-1 ; human immunodeficiency virus 1 ; SIV ; simian immunodeficiency virus ; SIVE ; SIV encephalitis ; AIDS ; acquired immune deficiency syndrome ; CHIT1 ; Chitotriosidase ; CHIA ; acidic mammalian chitinase ; CHI3L1 ; BRP-39 ; chitinase 3-like-1 ; CHI3L2 ; chitinase 3-like-2 ; CHID1 ; chitinase domain&ndash ; containing 1 ; CNS ; central nervous system ; CSF ; cerebrospinal fluid ; SMOX ; spermine oxidase ; DCBLD2 ; discoidin ; CUB and LCCL domain containing 2 ; LDLRAD4 ; low density lipoprotein receptor class A domain con

刊名：Virus Research

出版年：2017

8.How our approach for Sanfilippo syndrome could be a prototype for industrializing rare disease drug discovery and development

刊名：Molecular Genetics and Metabolism

出版年：2017

9.AAAV5-mediated gene therapy with choroid plexus-directed α-n-acetyl-glucosaminidase expression in Sanfilippo syndrome type B mice

刊名：Molecular Genetics and Metabolism

出版年：2017

10.Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice

作者：Shih-hsin Kan^a ; Steven Q. Le^a ; Quang D. Bui^a ; Braeden Benedict^a ; Jesse Cushman^b ; Mark S. Sands^c ; Patricia I. Dickson^a ; ^{pdickson@labiomed.org" class="auth_mail" title="E-mail the corresponding author}

关键词：AChE ; acetylcholinesterase ; ChAT ; choline acetyltransferase ; Naglu ; alpha-N-acetylglucosaminidase

刊名：Behavioural Brain Research

出版年：2016

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