Detalhe da pesquisa
1.
Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation.
J Biol Chem
; 299(12): 105475, 2023 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-37981208
2.
AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington's disease mouse model.
Brain
; 146(6): 2298-2315, 2023 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36508327
3.
Genetic topography and cortical cell loss in Huntington's disease link development and neurodegeneration.
Brain
; 146(11): 4532-4546, 2023 11 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-37587097
4.
PIAS1 modulates striatal transcription, DNA damage repair, and SUMOylation with relevance to Huntington's disease.
Proc Natl Acad Sci U S A
; 118(4)2021 01 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-33468657
5.
FTD-associated behavioural and transcriptomic abnormalities in 'humanized' progranulin-deficient mice: A novel model for progranulin-associated FTD.
Neurobiol Dis
; 182: 106138, 2023 06 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-37105261
6.
A novel mouse model for pyridoxine-dependent epilepsy due to antiquitin deficiency.
Hum Mol Genet
; 29(19): 3266-3284, 2020 11 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-32969477
7.
Targeting Huntingtin Expression in Patients with Huntington's Disease.
N Engl J Med
; 380(24): 2307-2316, 2019 06 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31059641
8.
Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components.
Mol Pharm
; 19(6): 1669-1686, 2022 06 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35594500
9.
Human progranulin-expressing mice as a novel tool for the development of progranulin-modulating therapeutics.
Neurobiol Dis
; 153: 105314, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33636385
10.
Mutant huntingtin expression in microglia is neither required nor sufficient to cause the Huntington's disease-like phenotype in BACHD mice.
Hum Mol Genet
; 28(10): 1661-1670, 2019 05 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30624705
11.
Tominersen in Adults with Manifest Huntington's Disease.
N Engl J Med
; 389(23): 2203-2205, 2023 Dec 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-38055260
12.
Composite UHDRS Correlates With Progression of Imaging Biomarkers in Huntington's Disease.
Mov Disord
; 36(5): 1259-1264, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33471951
13.
Longitudinal Structural MRI in Neurologically Healthy Adults.
J Magn Reson Imaging
; 52(5): 1385-1399, 2020 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-32469154
14.
KEAP1-modifying small molecule reveals muted NRF2 signaling responses in neural stem cells from Huntington's disease patients.
Proc Natl Acad Sci U S A
; 114(23): E4676-E4685, 2017 06 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28533375
15.
Testing a longitudinal compensation model in premanifest Huntington's disease.
Brain
; 141(7): 2156-2166, 2018 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29788038
16.
DNA methylation profiling in human Huntington's disease brain.
Hum Mol Genet
; 25(10): 2013-2030, 2016 05 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26953320
17.
Conditional loss of progranulin in neurons is not sufficient to cause neuronal ceroid lipofuscinosis-like neuropathology in mice.
Neurobiol Dis
; 106: 14-22, 2017 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-28647554
18.
Structural and functional brain network correlates of depressive symptoms in premanifest Huntington's disease.
Hum Brain Mapp
; 38(6): 2819-2829, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28294457
19.
Selective depletion of microglial progranulin in mice is not sufficient to cause neuronal ceroid lipofuscinosis or neuroinflammation.
J Neuroinflammation
; 14(1): 225, 2017 Nov 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-29149899
20.
Design optimization for clinical trials in early-stage manifest Huntington's disease.
Mov Disord
; 32(11): 1610-1619, 2017 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-28906031