Detalhe da pesquisa
1.
Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice.
Blood
; 141(19): 2316-2329, 2023 05 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36790505
2.
Region-Specific KCC2 Rescue by rhIGF-1 and Oxytocin in a Mouse Model of Rett Syndrome.
Cereb Cortex
; 32(13): 2885-2894, 2022 06 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-34791112
3.
Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery.
Hum Mol Genet
; 27(5): 761-779, 2018 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29281027
4.
AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy.
Mol Ther
; 25(12): 2727-2742, 2017 Dec 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28882452
5.
New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets.
iScience
; 27(5): 109777, 2024 May 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-38711458
6.
MCT1-dependent energetic failure and neuroinflammation underlie optic nerve degeneration in Wolfram syndrome mice.
Elife
; 122023 01 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-36645345
7.
Genome-wide screening in pluripotent cells identifies Mtf1 as a suppressor of mutant huntingtin toxicity.
Nat Commun
; 14(1): 3962, 2023 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-37407555
8.
Scn1a gene reactivation after symptom onset rescues pathological phenotypes in a mouse model of Dravet syndrome.
Nat Commun
; 13(1): 161, 2022 01 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35013317
9.
Modeling native and seeded Synuclein aggregation and related cellular dysfunctions in dopaminergic neurons derived by a new set of isogenic iPSC lines with SNCA multiplications.
Cell Death Dis
; 13(10): 881, 2022 10 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-36261424
10.
SETBP1 accumulation induces P53 inhibition and genotoxic stress in neural progenitors underlying neurodegeneration in Schinzel-Giedion syndrome.
Nat Commun
; 12(1): 4050, 2021 06 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-34193871
11.
Microglia-specific overexpression of α-synuclein leads to severe dopaminergic neurodegeneration by phagocytic exhaustion and oxidative toxicity.
Nat Commun
; 12(1): 6237, 2021 10 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-34716339
12.
Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome.
Elife
; 92020 03 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-32207685
13.
Frataxin gene editing rescues Friedreich's ataxia pathology in dorsal root ganglia organoid-derived sensory neurons.
Nat Commun
; 11(1): 4178, 2020 08 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-32826895
14.
Recipes for Making Neurons using Combinatorial Forward Genetics.
Cell Stem Cell
; 23(1): 13-14, 2018 07 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-29979987
15.
Rapid and efficient CRISPR/Cas9 gene inactivation in human neurons during human pluripotent stem cell differentiation and direct reprogramming.
Sci Rep
; 6: 37540, 2016 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-27857203
16.
Author Correction: Microglia-specific overexpression of α-synuclein leads to severe dopaminergic neurodegeneration by phagocytic exhaustion and oxidative toxicity.
Nat Commun
; 12(1): 7359, 2021 Dec 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-34916524