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Cholesterol Redistribution Triggered by Cyp46A1 Gene Therapy Improves Major Hallmarks of Niemann-Pick Type C Disease but Is Not Sufficient to Halt Neurodegeneration

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P-00Z-RDF

Author(s)

Nunes, MJ

Carvalho, AN

Reis, J

Costa, D

Moutinho, M

Mateus, J

de Almeida, RM

Brito, S

Risso, D

Nunes, S

Castro-Caldas, M

Gama, MJ

Rodrigues, CMP

Xapelli, S

Diógenes, MJ

Cartier, N

Chali, F

Piguet, F

Rodrigues, E

Tipo de Documento

Article

Year published

2024

Publicado

in BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE, ISSN: 0925-4439

Volume: 1870, Número: 3, Páginas: 166993 (22)

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Publication Identifiers

DOI: 10.1016/j.bbadis.2023.166993

Pubmed: 38142760

SCOPUS: 2-s2.0-85181842546

Wos: WOS:001153521200001

Source Identifiers

ISSN: 0925-4439

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Cholesterol Redistribution Triggered by Cyp46A1 Gene Therapy Improves Major Hallmarks of Niemann-Pick Type C Disease but Is Not Sufficient to Halt Neurodegeneration

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Cholesterol Redistribution Triggered by Cyp46A1 Gene Therapy Improves Major Hallmarks of Niemann-Pick Type C Disease but Is Not Sufficient to Halt Neurodegeneration

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