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1.
Genome Editing Tools for Lysosomal Storage Disorders.
Adv Exp Med Biol
; 1429: 127-155, 2023.
Article
in English
| MEDLINE | ID: mdl-37486520
2.
Protective effects of L-carnitine on behavioral alterations and neuroinflammation in striatum of glutaryl-COA dehydrogenase deficient mice.
Arch Biochem Biophys
; 709: 108970, 2021 09 30.
Article
in English
| MEDLINE | ID: mdl-34181873
3.
Cardiac pathology in mucopolysaccharidosis I mice: Losartan modifies ERK1/2 activation during cardiac remodeling.
J Inherit Metab Dis
; 44(3): 740-750, 2021 05.
Article
in English
| MEDLINE | ID: mdl-33145772
4.
Preliminary results of PBA-loaded nanoparticles development and the effect on oxidative stress and neuroinflammation in rats submitted to a chemically induced chronic model of MSUD.
Metab Brain Dis
; 36(5): 1015-1027, 2021 06.
Article
in English
| MEDLINE | ID: mdl-33620579
5.
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice.
Gene Ther
; 27(1-2): 74-84, 2020 02.
Article
in English
| MEDLINE | ID: mdl-31827259
6.
Damage-associated molecular patterns (DAMPs) related to immunogenic cell death are differentially triggered by clinically relevant chemotherapeutics in lung adenocarcinoma cells.
BMC Cancer
; 20(1): 474, 2020 May 26.
Article
in English
| MEDLINE | ID: mdl-32456685
7.
Estimated birth prevalence of mucopolysaccharidoses in Brazil.
Am J Med Genet A
; 182(3): 469-483, 2020 03.
Article
in English
| MEDLINE | ID: mdl-31926052
8.
Cathepsin B-associated Activation of Amyloidogenic Pathway in Murine Mucopolysaccharidosis Type I Brain Cortex.
Int J Mol Sci
; 21(4)2020 Feb 20.
Article
in English
| MEDLINE | ID: mdl-32093427
9.
Characterization of heart disease in mucopolysaccharidosis type II mice.
Cardiovasc Pathol
; 67: 107575, 2023.
Article
in English
| MEDLINE | ID: mdl-37730078
10.
Losartan improves aortic dilatation and cardiovascular disease in mucopolysaccharidosis I.
J Inherit Metab Dis
; 40(3): 311-312, 2017 05.
Article
in English
| MEDLINE | ID: mdl-28160112
11.
Network Analysis Reveals Proteins Associated with Aortic Dilatation in Mucopolysaccharidoses.
Interdiscip Sci
; 13(1): 34-43, 2021 Mar.
Article
in English
| MEDLINE | ID: mdl-33475959
12.
Genome editing in lysosomal disorders.
Prog Mol Biol Transl Sci
; 182: 289-325, 2021.
Article
in English
| MEDLINE | ID: mdl-34175045
13.
In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system.
J Control Release
; 288: 23-33, 2018 10 28.
Article
in English
| MEDLINE | ID: mdl-30170069
14.
Cathepsin B inhibition attenuates cardiovascular pathology in mucopolysaccharidosis I mice.
Life Sci
; 196: 102-109, 2018 Mar 01.
Article
in English
| MEDLINE | ID: mdl-29366749
15.
Gene Therapy for Lysosomal Storage Disorders: Recent Advances and Limitations
J. inborn errors metab. screen
; 5: e160028, 2017. tab
Article
in English
|
LILACS-Express
| ID: biblio-1090937
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