Detalhe da pesquisa
1.
Intrathecal Gene Therapy for Giant Axonal Neuropathy.
N Engl J Med
; 390(12): 1092-1104, 2024 03 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-38507752
2.
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy.
Gene Ther
; 31(5-6): 234-241, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38135787
3.
The diversity bonus in pooling local knowledge about complex problems.
Proc Natl Acad Sci U S A
; 118(5)2021 02 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33495329
4.
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Mol Ther
; 30(7): 2416-2428, 2022 07 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35585789
5.
Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis.
Int J Mol Sci
; 24(19)2023 Sep 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-37834060
6.
Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis.
Int J Mol Sci
; 24(11)2023 May 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-37298170
7.
Influence of institutional attributes on fulfillment of transfer and breeding recommendations in zoos and aquariums.
Zoo Biol
; 42(1): 142-149, 2023 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-35960010
8.
Institutional networks in cooperative population management: Exploring patterns in transfer fulfillment.
Zoo Biol
; 42(4): 547-556, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36872655
9.
Expression of phosphorylated ribosomal protein S6 in mesothelioma patients - correlation with clinico-pathological characteristics and outcome: results from the European Thoracic Oncology Platform (ETOP) Mesoscape project.
Mod Pathol
; 35(12): 1888-1899, 2022 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-36115922
10.
Analysis of new treatments proposed for malignant pleural mesothelioma raises concerns about the conduction of clinical trials in oncology.
J Transl Med
; 20(1): 593, 2022 12 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-36514092
11.
Analytical Validation of a Novel 6-Gene Signature for Prediction of Distant Recurrence in Estrogen Receptor-Positive, HER2-Negative, Early-Stage Breast Cancer.
Clin Chem
; 68(6): 837-847, 2022 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35312747
12.
Engineered microRNA-based regulatory element permits safe high-dose miniMECP2 gene therapy in Rett mice.
Brain
; 144(10): 3005-3019, 2021 11 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-33950254
13.
Giant axonal neuropathy: cross-sectional analysis of a large natural history cohort.
Brain
; 144(10): 3239-3250, 2021 11 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-34114613
14.
Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation.
Mol Ther
; 29(3): 989-1000, 2021 03 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33186692
15.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
16.
Altered expression of ACOX2 in non-small cell lung cancer.
BMC Pulm Med
; 22(1): 321, 2022 Aug 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-35999530
17.
Reasons for unfulfilled breeding and transfer recommendations in zoos and aquariums.
Zoo Biol
; 41(2): 143-156, 2022 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-34870877
18.
Viral-based rodent and nonhuman primate models of multiple system atrophy: Fidelity to the human disease.
Neurobiol Dis
; 148: 105184, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33221532
19.
Ethical challenges for a new generation of early-phase pediatric gene therapy trials.
Genet Med
; 23(11): 2057-2066, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34234300
20.
Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease.
Exp Eye Res
; 207: 108600, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33930398