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1.
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
Mol Ther
; 30(1): 145-163, 2022 01 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-34418541
2.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human ß-globin locus.
Blood
; 131(17): 1960-1973, 2018 04 26.
Artículo
en Inglés
| MEDLINE | ID: mdl-29519807
3.
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
Mol Ther
; 27(1): 137-150, 2019 01 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-30424953
4.
High-mobility group box-1 protein and ß-amyloid oligomers promote neuronal differentiation of adult hippocampal neural progenitors via receptor for advanced glycation end products/nuclear factor-κB axis: relevance for Alzheimer's disease.
J Neurosci
; 33(14): 6047-59, 2013 Apr 03.
Artículo
en Inglés
| MEDLINE | ID: mdl-23554486
5.
The noradrenergic component in tapentadol action counteracts µ-opioid receptor-mediated adverse effects on adult neurogenesis.
Mol Pharmacol
; 85(5): 658-70, 2014 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-24516101
6.
α2δ ligands act as positive modulators of adult hippocampal neurogenesis and prevent depression-like behavior induced by chronic restraint stress.
Mol Pharmacol
; 82(2): 271-80, 2012 Aug.
Artículo
en Inglés
| MEDLINE | ID: mdl-22572885
7.
Intracerebroventricular transplantation of human iPSC-derived neural stem cells (hiPSC-NSCs) into neonatal mice.
Methods Cell Biol
; 171: 127-147, 2022.
Artículo
en Inglés
| MEDLINE | ID: mdl-35953197
8.
A novel role for the Receptor for Advanced Glycation End-products in neural progenitor cells derived from adult SubVentricular Zone.
Mol Cell Neurosci
; 45(2): 139-50, 2010 Oct.
Artículo
en Inglés
| MEDLINE | ID: mdl-20600932
9.
In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges.
Front Med (Lausanne)
; 8: 774618, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-35118085
10.
Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System.
Front Genome Ed
; 3: 644319, 2021.
Artículo
en Inglés
| MEDLINE | ID: mdl-34713256
11.
Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases.
Front Mol Biosci
; 7: 224, 2020.
Artículo
en Inglés
| MEDLINE | ID: mdl-33062642
12.
GATA Factor-Mediated Gene Regulation in Human Erythropoiesis.
iScience
; 23(4): 101018, 2020 Apr 24.
Artículo
en Inglés
| MEDLINE | ID: mdl-32283524
13.
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Sci Adv
; 6(7)2020 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-32917636
14.
Editorial: Ex-vivo and in-vivo genome engineering for metabolic and neurometabolic diseases.
Front Genome Ed
; 5: 1248904, 2023.
Artículo
en Inglés
| MEDLINE | ID: mdl-37484653
15.
Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy.
Cell Death Dis
; 9(6): 698, 2018 06 13.
Artículo
en Inglés
| MEDLINE | ID: mdl-29899471
16.
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.
Mol Ther Methods Clin Dev
; 10: 268-280, 2018 Sep 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-30140714
17.
Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.
Stem Cells Transl Med
; 6(2): 352-368, 2017 02.
Artículo
en Inglés
| MEDLINE | ID: mdl-28191778
18.
Novel TBX3 mutation data in families with ulnar-mammary syndrome indicate a genotype-phenotype relationship: mutations that do not disrupt the T-domain are associated with less severe limb defects.
Eur J Med Genet
; 49(2): 151-8, 2006.
Artículo
en Inglés
| MEDLINE | ID: mdl-16530712
19.
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy.
EMBO Mol Med
; 8(5): 489-510, 2016 05.
Artículo
en Inglés
| MEDLINE | ID: mdl-27025653
20.
TBX3, the gene mutated in ulnar-mammary syndrome, promotes growth of mammary epithelial cells via repression of p19ARF, independently of p53.
Cell Tissue Res
; 328(2): 301-16, 2007 May.
Artículo
en Inglés
| MEDLINE | ID: mdl-17265068
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