Detalhe da pesquisa
1.
The multifaceted roles of the brain glycogen.
J Neurochem
; 2023 Aug 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37554056
2.
Current avenues of gene therapy in Pompe disease.
Curr Opin Neurol
; 36(5): 464-473, 2023 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37639402
3.
Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study).
Ann Neurol
; 89(2): 212-225, 2021 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33068037
4.
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
5.
Gauging Gait Disorders with a Method Inspired by Motor Control Theories: A Pilot Study in Friedreich's Ataxia.
Sensors (Basel)
; 21(4)2021 Feb 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33562027
6.
Temporal but not spatial dysmetria relates to disease severity in FA.
J Neurophysiol
; 123(2): 718-725, 2020 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31693434
7.
Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy.
Mol Ther
; 30(12): 3503-3504, 2022 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-36436512
8.
Considerations for Systemic Use of Gene Therapy.
Mol Ther
; 29(2): 422-423, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33485465
9.
Scratching the surface of RGD-directed AAV capsid engineering.
Mol Ther
; 29(11): 3099-3100, 2021 11 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34699781
10.
Respiratory motor function in individuals with centronuclear myopathies.
Muscle Nerve
; 53(2): 214-21, 2016 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-26351754
11.
Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction.
Muscle Nerve
; 51(6): 877-83, 2015 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-25186912
12.
Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapies.
Pediatr Pulmonol
; 59(5): 1364-1371, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38358081
13.
Perspectives of the Friedreich ataxia community on gene therapy clinical trials.
Mol Ther Methods Clin Dev
; 32(1): 101179, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38261944
14.
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.
Mol Ther Methods Clin Dev
; 32(1): 101216, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38440160
15.
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
J Clin Invest
; 134(1)2024 Jan 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-37988172
16.
Evolving Horizons: Adenovirus Vectors' Timeless Influence on Cancer, Gene Therapy and Vaccines.
Viruses
; 15(12)2023 12 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38140619
17.
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation.
Mol Ther Methods Clin Dev
; 30: 303-314, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37601414
18.
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Sci Transl Med
; 15(677): eabo1815, 2023 01 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36599002
19.
Natural History of Friedreich's Ataxia: Heterogeneity of Neurological Progression and Consequences for Clinical Trial Design.
Neurology
; 2022 Jul 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35817567
20.
A natural history study to track brain and spinal cord changes in individuals with Friedreich's ataxia: TRACK-FA study protocol.
PLoS One
; 17(11): e0269649, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-36410013