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.
Glycogen synthase downregulation rescues the amylopectinosis of murine RBCK1 deficiency.
Brain
; 145(7): 2361-2377, 2022 07 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-35084461
6.
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.
Nucleic Acids Res
; 49(2): 657-673, 2021 01 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-33367834
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.
Gys1 antisense therapy rescues neuropathological bases of murine Lafora disease.
Brain
; 144(10): 2985-2993, 2021 11 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-33993268
9.
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
10.
Convective forces increase rostral delivery of intrathecal radiotracers and antisense oligonucleotides in the cynomolgus monkey nervous system.
J Transl Med
; 18(1): 309, 2020 08 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-32771027
11.
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
12.
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
13.
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
14.
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
15.
Intrathecal administration of antisense oligonucleotide against p38α but not p38ß MAP kinase isoform reduces neuropathic and postoperative pain and TLR4-induced pain in male mice.
Brain Behav Immun
; 72: 34-44, 2018 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-29128611
16.
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
17.
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
18.
Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.
Nucleic Acids Res
; 41(21): 9634-50, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23963702
19.
Totally tubular: ASO-mediated knock-down of G2019S -Lrrk2 modulates lysosomal tubule-associated antigen presentation in macrophages.
bioRxiv
; 2023 Jul 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-37503274
20.
ASO-mediated knockdown or kinase inhibition of G2019S-Lrrk2 modulates lysosomal tubule-associated antigen presentation in macrophages.
Mol Ther Nucleic Acids
; 34: 102064, 2023 Dec 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-38028198