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1.
Further Delineation of Duplications of ARX Locus Detected in Male Patients with Varying Degrees of Intellectual Disability.
Int J Mol Sci
; 23(6)2022 Mar 13.
Artículo
en Inglés
| MEDLINE | ID: mdl-35328505
2.
Histone demethylase KDM5C is a SAHA-sensitive central hub at the crossroads of transcriptional axes involved in multiple neurodevelopmental disorders.
Hum Mol Genet
; 28(24): 4089-4102, 2019 12 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-31691806
3.
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.
Cell Rep Med
; : 101619, 2024 Jun 11.
Artículo
en Inglés
| MEDLINE | ID: mdl-38897206
4.
Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease.
JCI Insight
; 8(21)2023 Nov 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-37707949
5.
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice.
Mol Ther Methods Clin Dev
; 26: 495-504, 2022 Sep 08.
Artículo
en Inglés
| MEDLINE | ID: mdl-36092366
6.
Analysis of a Set of KDM5C Regulatory Genes Mutated in Neurodevelopmental Disorders Identifies Temporal Coexpression Brain Signatures.
Genes (Basel)
; 12(7)2021 07 18.
Artículo
en Inglés
| MEDLINE | ID: mdl-34356104
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