Detalhe da pesquisa
1.
Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.
Cell
; 150(5): 895-908, 2012 Aug 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-22939619
2.
A single-cell map of antisense oligonucleotide activity in the brain.
Nucleic Acids Res
; 51(14): 7109-7124, 2023 08 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37188501
3.
Antisense Drugs Make Sense for Neurological Diseases.
Annu Rev Pharmacol Toxicol
; 61: 831-852, 2021 01 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33035446
4.
Alternative processing of human HTT mRNA with implications for Huntington's disease therapeutics.
Brain
; 145(12): 4409-4424, 2022 12 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-35793238
5.
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
6.
Prion protein lowering is a disease-modifying therapy across prion disease stages, strains and endpoints.
Nucleic Acids Res
; 48(19): 10615-10631, 2020 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32776089
7.
A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.
Hum Mol Genet
; 26(6): 1115-1132, 2017 03 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28104789
8.
Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.
Ann Neurol
; 84(1): 64-77, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29908063
9.
MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.
J Neurosci
; 37(22): 5574-5586, 2017 05 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-28416596
10.
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.
Hum Mol Genet
; 25(R1): R53-64, 2016 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26503961
11.
ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43.
Proc Natl Acad Sci U S A
; 110(8): E736-45, 2013 Feb 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-23382207
12.
In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.
Mol Ther
; 22(12): 2093-2106, 2014 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-25101598
13.
RNA-targeted therapy corrects neuronal deficits in PACS1 syndrome mice.
Res Sq
; 2023 Jan 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36747781
14.
A single-cell map of antisense oligonucleotide activity in the brain.
bioRxiv
; 2023 Feb 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-36824749
15.
Neural deficits in a mouse model of PACS1 syndrome are corrected with PACS1- or HDAC6-targeting therapy.
Nat Commun
; 14(1): 6547, 2023 10 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-37848409
16.
Regional variability and genotypic and pharmacodynamic effects on PrP concentration in the CNS.
JCI Insight
; 7(6)2022 03 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35133987
17.
Erratum: LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson's Disease Mouse Model.
Mol Ther Nucleic Acids
; 25: 152-154, 2021 Sep 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34458000
18.
Erratum: LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson's Disease Mouse Model.
Mol Ther Nucleic Acids
; 24: 1051-1053, 2021 Jun 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-34141459
19.
α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease.
JCI Insight
; 6(5)2021 03 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-33682798
20.
Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks.
JAMA Neurol
; 77(6): 764-772, 2020 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32202594