Detalhe da pesquisa
1.
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Mol Ther
; 31(9): 2767-2782, 2023 09 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37481701
2.
Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart Failure-ANVIAS Study.
Int J Mol Sci
; 24(6)2023 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-36982654
3.
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease.
Mol Ther
; 29(1): 162-175, 2021 01 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33010819
4.
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
N Engl J Med
; 377(18): 1713-1722, 2017 11 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29091557
5.
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Mol Ther
; 27(10): 1836-1847, 2019 10 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-31331814
6.
Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism.
Proc Natl Acad Sci U S A
; 113(42): E6496-E6505, 2016 10 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-27688759
7.
Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.
Proc Natl Acad Sci U S A
; 111(2): 829-32, 2014 Jan 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-24379375
8.
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Mol Ther
; 23(3): 477-87, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25358252
9.
Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex.
J Neurosci
; 34(47): 15587-600, 2014 Nov 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-25411487
10.
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.
Mol Ther
; 21(12): 2148-59, 2013 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24008656
11.
AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.
Mol Ther Methods Clin Dev
; 32(1): 101176, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38225934
12.
In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders.
Biology (Basel)
; 12(6)2023 Jun 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-37372153
13.
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.
Mol Ther Methods Clin Dev
; 30: 16-29, 2023 Sep 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-37746244
14.
Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.
Front Genet
; 14: 1118649, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37035740
15.
Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8mnd mice.
Orphanet J Rare Dis
; 17(1): 411, 2022 11 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-36369162
16.
AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency.
Thyroid
; 32(7): 849-859, 2022 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35350867
17.
Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes.
Cells
; 11(7)2022 04 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35406750
18.
Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV.
Cell Rep Methods
; 2(6): 100236, 2022 06 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-35784645
19.
Mechanisms of IRF2BPL-related disorders and identification of a potential therapeutic strategy.
Cell Rep
; 41(10): 111751, 2022 Dec 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36476864
20.
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
Mol Ther Methods Clin Dev
; 20: 497-507, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33665223