Detalhe da pesquisa
1.
Regulated control of gene therapies by drug-induced splicing.
Nature
; 596(7871): 291-295, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34321659
2.
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease.
Mol Ther
; 32(5): 1373-1386, 2024 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38504517
3.
Immortalized striatal precursor neurons from Huntington's disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics.
Hum Mol Genet
; 30(24): 2469-2487, 2021 11 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-34296279
4.
Author Correction: Regulated control of gene therapies by drug-induced splicing.
Nature
; 602(7897): E25, 2022 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-35105975
5.
Targeted Allele Suppression Prevents Progressive Hearing Loss in the Mature Murine Model of Human TMC1 Deafness.
Mol Ther
; 27(3): 681-690, 2019 03 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30686588
6.
RNA Interference Prevents Autosomal-Dominant Hearing Loss.
Am J Hum Genet
; 98(6): 1101-1113, 2016 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-27236922
7.
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates.
Mol Ther
; 31(3): 609-612, 2023 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36610400
8.
HOMER2, a stereociliary scaffolding protein, is essential for normal hearing in humans and mice.
PLoS Genet
; 11(3): e1005137, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25816005
9.
Comprehensive genetic testing in the clinical evaluation of 1119 patients with hearing loss.
Hum Genet
; 135(4): 441-450, 2016 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26969326
10.
Mutation-agnostic RNA interference with engineered replacement rescues Tmc1-related hearing loss.
Life Sci Alliance
; 6(3)2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36574989
11.
Single-cell RNA-sequencing of stria vascularis cells in the adult Slc26a4-/- mouse.
BMC Med Genomics
; 16(1): 133, 2023 06 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-37322474
12.
APOE2 gene therapy reduces amyloid deposition, and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease.
bioRxiv
; 2023 Aug 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-37645718
13.
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF.
Mol Ther Nucleic Acids
; 33: 296-304, 2023 Sep 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37547292
14.
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching.
Nat Commun
; 14(1): 2628, 2023 05 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37149717
15.
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain.
Nat Med
; 27(11): 1982-1989, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34663988
16.
Insights into the Biology of Hearing and Deafness Revealed by Single-Cell RNA Sequencing.
Cell Rep
; 26(11): 3160-3171.e3, 2019 03 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30865901
17.
Enhanced viral-mediated cochlear gene delivery in adult mice by combining canal fenestration with round window membrane inoculation.
Sci Rep
; 8(1): 2980, 2018 02 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-29445157
18.
Intravenous rAAV2/9 injection for murine cochlear gene delivery.
Sci Rep
; 7(1): 9609, 2017 08 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-28852025
19.
CDKL5 regulates flagellar length and localizes to the base of the flagella in Chlamydomonas.
Mol Biol Cell
; 24(5): 588-600, 2013 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-23283985