Detalhe da pesquisa
1.
SCA7 Mouse Cerebellar Pathology Reveals Preferential Downregulation of Key Purkinje Cell-Identity Genes and Shared Disease Signature with SCA1 and SCA2.
J Neurosci
; 41(22): 4910-4936, 2021 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33888607
2.
Rapid and Complete Reversal of Sensory Ataxia by Gene Therapy in a Novel Model of Friedreich Ataxia.
Mol Ther
; 26(8): 1940-1952, 2018 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29853274
3.
CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington's disease.
Brain
; 139(Pt 3): 953-70, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26912634
4.
DDIT4/REDD1/RTP801 is a novel negative regulator of Schwann cell myelination.
J Neurosci
; 33(38): 15295-305, 2013 Sep 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-24048858
5.
Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy.
Mol Ther Methods Clin Dev
; 32(2): 101248, 2024 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-38680552
6.
Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration.
Biochim Biophys Acta Mol Basis Dis
; 1870(3): 166993, 2024 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38142760
7.
Gene therapy for neurodegenerative disorders in children: dreams and realities.
Arch Pediatr
; 30(8S1): 8S32-8S40, 2023 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-38043981
8.
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.
Hum Mol Genet
; 19(1): 147-58, 2010 Jan 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-19837699
9.
AAV-delivered diacylglycerol kinase DGKk achieves long-term rescue of fragile X syndrome mouse model.
EMBO Mol Med
; 14(5): e14649, 2022 05 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35373916
10.
Bioelectronic cell-based device provides a strategy for the treatment of the experimental model of multiple sclerosis.
J Control Release
; 352: 994-1008, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36370877
11.
Optimized Protocol for Subcutaneous Implantation of Encapsulated Cells Device and Evaluation of Biocompatibility.
Front Bioeng Biotechnol
; 9: 620967, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34249877
12.
Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice.
Front Mol Neurosci
; 14: 677895, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34093126
13.
The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System.
Hum Gene Ther
; 32(7-8): 349-374, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33167739
14.
[Deglutition disorder: what to do?]. / Troubles de la déglutition: que faire?
Rev Med Suisse
; 11(459): 248, 2015 Jan 28.
Artigo
em Francês
| MEDLINE | ID: mdl-25845161
15.
Optimizing PCR for Mouse Genotyping: Recommendations for Reliable, Rapid, Cost Effective, Robust and Adaptable to High-Throughput Genotyping Protocol for Any Type of Mutation.
Curr Protoc Mouse Biol
; 9(4): e65, 2019 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-31756054
16.
Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain.
Mol Ther Methods Clin Dev
; 14: 237-251, 2019 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31440523
17.
Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future.
Hum Gene Ther
; 28(11): 988-1003, 2017 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29035118
18.
Niacin-mediated Tace activation ameliorates CMT neuropathies with focal hypermyelination.
EMBO Mol Med
; 8(12): 1438-1454, 2016 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-27799291
19.
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.
Hum Gene Ther Clin Dev
; 26(2): 113-24, 2015 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-25758611
20.
Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.
Hum Gene Ther
; 23(8): 903-14, 2012 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-22642214