Detalhe da pesquisa
1.
AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.
Mol Ther
; 28(8): 1887-1901, 2020 08 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32470325
2.
Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice.
Hum Mol Genet
; 27(6): 954-968, 2018 03 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-29325092
3.
Cofilin-1 phosphorylation catalyzed by ERK1/2 alters cardiac actin dynamics in dilated cardiomyopathy caused by lamin A/C gene mutation.
Hum Mol Genet
; 27(17): 3060-3078, 2018 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29878125
4.
A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.
Mol Ther
; 25(9): 2038-2052, 2017 09 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28663100
5.
The autophagy/lysosome pathway is impaired in SCA7 patients and SCA7 knock-in mice.
Acta Neuropathol
; 128(5): 705-22, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-24859968
6.
Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.
Mol Ther
; 21(2): 282-90, 2013 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-23295949
7.
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.
Hum Mol Genet
; 20(4): 681-93, 2011 Feb 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21118896
8.
Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription.
Sci Rep
; 12(1): 9674, 2022 06 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-35690627
9.
Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.
Front Genome Ed
; 4: 785698, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35359664
10.
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.
Mol Ther Methods Clin Dev
; 20: 1-17, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33335943
11.
The non-muscle ADF/cofilin-1 controls sarcomeric actin filament integrity and force production in striated muscle laminopathies.
Cell Rep
; 36(8): 109601, 2021 08 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-34433058
12.
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.
Mol Ther
; 17(7): 1187-96, 2009 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-19367261
13.
Activation of sarcolipin expression and altered calcium cycling in LMNA cardiomyopathy.
Biochem Biophys Rep
; 22: 100767, 2020 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-32490213
14.
Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.
Thromb Haemost
; 119(12): 1956-1967, 2019 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-31659733
15.
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins.
Mol Neurodegener
; 11(1): 58, 2016 07 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-27465358
16.
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Mol Ther Methods Clin Dev
; 3: 16060, 2016.
Artigo
em Inglês
| MEDLINE | ID: mdl-27652289
17.
Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice.
Front Mol Neurosci
; 8: 36, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26283910
18.
scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease.
Hum Gene Ther
; 24(7): 670-82, 2013 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-23799774
19.
Expression of mdr1 is required for efficient long term regeneration of dystrophic muscle.
Exp Cell Res
; 313(11): 2438-50, 2007 Jul 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-17481607
20.
Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb.
J Gene Med
; 7(6): 782-91, 2005 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-15693034