Detalhe da pesquisa
1.
Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis.
Brain
; 145(2): 655-669, 2022 04 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34410345
2.
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
Mol Ther
; 28(10): 2150-2160, 2020 10 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32592687
3.
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Mol Ther
; 28(2): 411-421, 2020 02 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-31813800
4.
In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis.
Mol Ther
; 29(9): 2633-2634, 2021 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34433081
5.
Hepatic production of transthyretin L12P leads to intracellular lysosomal aggregates in a new somatic transgenic mouse model.
Biochim Biophys Acta
; 1832(8): 1183-93, 2013 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-23579071
6.
Increased Pre-Boundary Lengthening Does Not Enhance Implicit Intonational Phrase Perception in European Portuguese: An EEG Study.
Brain Sci
; 13(3)2023 Mar 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36979251
7.
Duration perception for visual stimuli is impaired in dyslexia but deficits in visual processing may not be the culprits.
Sci Rep
; 13(1): 12873, 2023 08 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-37553469
8.
Domain-general but not speech-specific auditory duration perception predicts pseudoword reading in adults.
Front Hum Neurosci
; 17: 1241589, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37780963
9.
Observed High Adherence to Recombinant Human Growth Hormone Treatment Using a Multi-Component Approach to Improve Adherence in Individuals with Growth Disorders.
Stud Health Technol Inform
; 294: 817-818, 2022 May 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-35612215
10.
Biopsychosocial Correlates of Quality of Life in Multiple Sclerosis Patients.
Int J Environ Res Public Health
; 19(21)2022 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36361311
11.
AAV gene therapy for Tay-Sachs disease.
Nat Med
; 28(2): 251-259, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-35145305
12.
Real-time MR tracking of AAV gene therapy with ßgal-responsive MR probe in a murine model of GM1-gangliosidosis.
Mol Ther Methods Clin Dev
; 23: 128-134, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34703836
13.
Carvedilol treatment reduces transthyretin deposition in a familial amyloidotic polyneuropathy mouse model.
Pharmacol Res
; 62(6): 514-22, 2010 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-20692343
14.
Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B.
Hum Gene Ther
; 31(1-2): 90-102, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31696742
15.
Anti-apoptotic treatment reduces transthyretin deposition in a transgenic mouse model of Familial Amyloidotic Polyneuropathy.
Biochim Biophys Acta
; 1782(9): 517-22, 2008 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-18572024
16.
Response to: Randomized, assessor-blinded trial comparing highly purified human menotropin and recombinant follicle-stimulating hormone in high responders undergoing intracytoplasmic sperm injection.
Fertil Steril
; 121(2): 358, 2024 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-37734649
17.
Author Correction: Duration perception for visual stimuli is impaired in dyslexia but deficits in visual processing may not be the culprits.
Sci Rep
; 13(1): 15128, 2023 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37704746
18.
Gene Therapy for Huntington's Disease: The Future Is in Gene Editing.
Hum Gene Ther
; 33(1-2): 12-13, 2022 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-35049370
19.
Biomarkers in the assessment of therapies for familial amyloidotic polyneuropathy.
Mol Med
; 13(11-12): 584-91, 2007.
Artigo
em Inglês
| MEDLINE | ID: mdl-17932549