Detalhe da pesquisa
1.
A Human Bi-specific Antibody against Zika Virus with High Therapeutic Potential.
Cell
; 171(1): 229-241.e15, 2017 Sep 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-28938115
2.
Selective homing of CAR-CIK cells to the bone marrow niche enhances control of the acute myeloid leukemia burden.
Blood
; 141(21): 2587-2598, 2023 05 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-36787509
3.
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
4.
Temporal manipulation of the Scn1a gene reveals its essential role in adult brain function.
Brain
; 147(4): 1216-1230, 2024 Apr 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-37812819
5.
Genetics and gene therapy in Dravet syndrome.
Epilepsy Behav
; 131(Pt B): 108043, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-34053869
6.
Zrf1 is required to establish and maintain neural progenitor identity.
Genes Dev
; 28(2): 182-97, 2014 Jan 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-24449271
7.
CRISPR/Cas9-Induced Inactivation of the Autism-Risk Gene setd5 Leads to Social Impairments in Zebrafish.
Int J Mol Sci
; 24(1)2022 Dec 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-36613611
8.
SULT4A1 Modulates Synaptic Development and Function by Promoting the Formation of PSD-95/NMDAR Complex.
J Neurosci
; 40(37): 7013-7026, 2020 09 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32801157
9.
In vivo CRISPRa decreases seizures and rescues cognitive deficits in a rodent model of epilepsy.
Brain
; 143(3): 891-905, 2020 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32129831
10.
dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice.
Mol Ther
; 28(1): 235-253, 2020 01 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31607539
11.
Glucocerebrosidase Gene Therapy Induces Alpha-Synuclein Clearance and Neuroprotection of Midbrain Dopaminergic Neurons in Mice and Macaques.
Int J Mol Sci
; 22(9)2021 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34062940
12.
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
13.
A novel POLR3A genotype leads to leukodystrophy type-7 in two siblings with unusually late age of onset.
BMC Neurol
; 20(1): 258, 2020 Jun 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-32600288
14.
Reply: Spatial and temporal manipulation of the Scn1a gene affect adult brain function.
Brain
; 147(4): e31-e33, 2024 Apr 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-38099567
15.
SCN1A/NaV 1.1 channelopathies: Mechanisms in expression systems, animal models, and human iPSC models.
Epilepsia
; 60 Suppl 3: S25-S38, 2019 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31904127
16.
PRRT2 controls neuronal excitability by negatively modulating Na+ channel 1.2/1.6 activity.
Brain
; 141(4): 1000-1016, 2018 04 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29554219
17.
mSEL-1L deficiency affects vasculogenesis and neural stem cell lineage commitment.
J Cell Physiol
; 233(4): 3152-3163, 2018 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28816361
18.
The endoplasmic reticulum-mitochondria interface is perturbed in PARK2 knockout mice and patients with PARK2 mutations.
Hum Mol Genet
; 25(14): 2972-2984, 2016 07 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27206984
19.
The Tbr2 Molecular Network Controls Cortical Neuronal Differentiation Through Complementary Genetic and Epigenetic Pathways.
Cereb Cortex
; 27(6): 3378-3396, 2017 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27600842
20.
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