Nanoparticles containing β‐cyclodextrin potentially useful for the treatment of Niemann‐Pick C-Reference-Cited by-同舟云学术

Nanoparticles containing β‐cyclodextrin potentially useful for the treatment of Niemann‐Pick C

Published:2020-01-13 Issue:3 Volume:43 Page:586-601
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:Jrnl of Inher Metab Disea

Author:

Donida Bruna¹,Raabe Marco²,Tauffner Bárbara³,Farias Marcelo A.⁴,Machado Andryele Z.⁵,Timm Fernanda⁵,Kessler Rejane G.⁵,Hammerschmidt Tatiane G.⁶,Reinhardt Luiza S.²,Brito Verônica B.²⁷,Portugal Rodrigo V.⁴,Bernardi Andressa⁸,Frozza Rudimar⁸,Moura Dinara J.²,Giugliani Roberto⁵⁹,Poletto Fernanda¹³,Vargas Carmen R.¹⁵

Affiliation:

1. Programa de Pós‐Graduação em Ciências Biológicas: Bioquímica Universidade Federal do Rio Grande do Sul Porto Alegre Brazil

2. Laboratório de Genética Toxicológica Universidade Federal de Ciências da Saúde de Porto Alegre Porto Alegre Brazil

3. Programa de Pós Graduação em Química Universidade Federal do Rio Grande do Sul Porto Alegre Brazil

4. Brazilian Nanotechnology National Laboratory (LNNano) Brazilian Center for Research in Energy and Materials (CNPEM) Campinas Brazil

5. Serviço de Genética Médica Hospital de Clínicas de Porto Alegre Porto Alegre Brazil

6. Programa de Pós Graduação em Ciências Farmacêuticas Universidade Federal do Rio Grande do Sul Porto Alegre Brazil

7. Departamento de Fisioterapia Faculdades Integradas de Taquara (FACCAT) Taquara Brazil

8. Instituto Oswaldo Cruz (IOC) Fundação Oswaldo Cruz (FIOCRUZ) Rio de Janeiro Brazil

9. Department of Genetics Universidade Federal do Rio Grande do Sul Porto Alegre Brazil

Funder

Conselho Nacional de Desenvolvimento Científico e Tecnológico

Fundação Carlos Chagas Filho de Amparo à Pesquisa do Estado do Rio de Janeiro

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12210

Reference57 articles.

1. High incidence of unrecognized visceral/neurological late-onset Niemann-Pick disease, type C1, predicted by analysis of massively parallel sequencing data sets

2. Niemann-Pick disease type C

3. Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport of cholesterol in Niemann-Pick type C cells

4. Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

5. Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin

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