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1.
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
Gene Ther;
30(5): 443-454, 2023 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-36450833
2.
Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice.
Gene Ther;
28(7-8): 456-468, 2021 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-33612827
3.
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice.
Gene Ther;
28(10-11): 646-658, 2021 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-33558692
4.
Development of an aggregate-selective, human-derived α-synuclein antibody BIIB054 that ameliorates disease phenotypes in Parkinson's disease models.
Neurobiol Dis;
124: 276-288, 2019 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-30381260
5.
Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection.
Mol Ther Nucleic Acids;
34: 102057, 2023 Dec 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-37928442
6.
RNA tape sampling in cutaneous lupus erythematosus discriminates affected from unaffected and healthy volunteer skin.
Lupus Sci Med;
8(1)2021 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-33658303
7.
Antisense Oligonucleotide-Mediated Reduction of HDAC6 Does Not Reduce Tau Pathology in P301S Tau Transgenic Mice.
Front Neurol;
12: 624051, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-34262517
8.
BIN1 protein isoforms are differentially expressed in astrocytes, neurons, and microglia: neuronal and astrocyte BIN1 are implicated in tau pathology.
Mol Neurodegener;
15(1): 44, 2020 07 29.
Artículo
en Inglés
| MEDLINE | ID: mdl-32727516
9.
Cell-autonomous and non-cell autonomous effects of neuronal BIN1 loss in vivo.
PLoS One;
14(8): e0220125, 2019.
Artículo
en Inglés
| MEDLINE | ID: mdl-31408457
10.
BIN1 favors the spreading of Tau via extracellular vesicles.
Sci Rep;
9(1): 9477, 2019 07 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-31263146
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