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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.
Article
in English
| MEDLINE | ID: mdl-37481701
2.
CuATSM effectively ameliorates ALS patient astrocyte-mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis.
Glia
; 71(2): 350-365, 2023 02.
Article
in English
| MEDLINE | ID: mdl-36213964
3.
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.
Article
in English
| MEDLINE | ID: mdl-36982654
4.
PolyGA targets the ER stress-adaptive response by impairing GRP75 function at the MAM in C9ORF72-ALS/FTD.
Acta Neuropathol
; 144(5): 939-966, 2022 11.
Article
in English
| MEDLINE | ID: mdl-36121477
5.
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.
Article
in English
| MEDLINE | ID: mdl-33010819
6.
Neurodegenerative Diseases: From Dysproteostasis, Altered Calcium Signalosome to Selective Neuronal Vulnerability to AAV-Mediated Gene Therapy.
Int J Mol Sci
; 23(22)2022 Nov 16.
Article
in English
| MEDLINE | ID: mdl-36430666
7.
Intellectual disability: dendritic anomalies and emerging genetic perspectives.
Acta Neuropathol
; 141(2): 139-158, 2021 02.
Article
in English
| MEDLINE | ID: mdl-33226471
8.
Mutations in glycyl-tRNA synthetase impair mitochondrial metabolism in neurons.
Hum Mol Genet
; 27(12): 2187-2204, 2018 06 15.
Article
in English
| MEDLINE | ID: mdl-29648643
9.
The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy.
EMBO J
; 35(15): 1656-76, 2016 08 01.
Article
in English
| MEDLINE | ID: mdl-27334615
10.
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
N Engl J Med
; 377(18): 1713-1722, 2017 11 02.
Article
in English
| MEDLINE | ID: mdl-29091557
11.
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Mol Ther
; 27(10): 1836-1847, 2019 10 02.
Article
in English
| MEDLINE | ID: mdl-31331814
12.
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.
Article
in English
| MEDLINE | ID: mdl-27688759
13.
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.
Article
in English
| MEDLINE | ID: mdl-24379375
14.
A large animal model of spinal muscular atrophy and correction of phenotype.
Ann Neurol
; 77(3): 399-414, 2015 Mar.
Article
in English
| MEDLINE | ID: mdl-25516063
15.
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.
Article
in English
| MEDLINE | ID: mdl-25358252
16.
Functional Capacity Evaluation: Performance of Patients with Chronic Non-specific Low Back Pain Without Waddell Signs.
J Occup Rehabil
; 25(2): 257-66, 2015 Jun.
Article
in English
| MEDLINE | ID: mdl-25190669
17.
Splicing changes in SMA mouse motoneurons and SMN-depleted neuroblastoma cells: evidence for involvement of splicing regulatory proteins.
RNA Biol
; 11(11): 1430-46, 2014.
Article
in English
| MEDLINE | ID: mdl-25692239
18.
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.
Article
in English
| MEDLINE | ID: mdl-24008656
19.
AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models.
Mol Ther Methods Clin Dev
; 32(1): 101176, 2024 Mar 14.
Article
in English
| MEDLINE | ID: mdl-38225934
20.
RNA helicase IGHMBP2 regulates THO complex to ensure cellular mRNA homeostasis.
Cell Rep
; 43(2): 113802, 2024 Feb 27.
Article
in English
| MEDLINE | ID: mdl-38368610