Detalhe da pesquisa
1.
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia.
J Allergy Clin Immunol
; 2024 Mar 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-38479630
2.
Dominant and recessive mutations in rhodopsin activate different cell death pathways.
Hum Mol Genet
; 25(13): 2801-2812, 2016 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27149983
3.
Correction of IVS I-110(G>A) ß-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
Haematologica
; 104(11): e497-e501, 2019 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-31004018
4.
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells.
Hum Gene Ther
; 35(7-8): 269-283, 2024 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-38251667
5.
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV.
Mol Ther Nucleic Acids
; 35(1): 102134, 2024 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-38384445
6.
Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Mol Ther Nucleic Acids
; 34: 102066, 2023 Dec 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-38034032
7.
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.
Mol Ther Methods Clin Dev
; 27: 96-108, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36212909
8.
Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq.
Cell Stem Cell
; 28(6): 1136-1147.e5, 2021 06 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33626327
9.
Gene Editing and Genotoxicity: Targeting the Off-Targets.
Front Genome Ed
; 2: 613252, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-34713236
10.
Correction: Genomic Analysis of Sleeping Beauty Transposon Integration in Human Somatic Cells.
PLoS One
; 15(1): e0228703, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-31999797
11.
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.
Nat Commun
; 11(1): 4034, 2020 08 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-32788576
12.
Pigment epithelium-derived factor hinders photoreceptor cell death by reducing intracellular calcium in the degenerating retina.
Cell Death Dis
; 9(5): 560, 2018 05 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29752430
13.
Genome and Epigenome Editing to Treat Disorders of the Hematopoietic System.
Hum Gene Ther
; 28(11): 1105-1115, 2017 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-28806883
14.
Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes.
J Invest Dermatol
; 137(4): 836-844, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-28027893
15.
Genomic analysis of Sleeping Beauty transposon integration in human somatic cells.
PLoS One
; 9(11): e112712, 2014.
Artigo
em Inglês
| MEDLINE | ID: mdl-25390293
16.
Failure to detect DNA-guided genome editing using Natronobacterium gregoryi Argonaute.
Nat Biotechnol
; 35(1): 17-18, 2016 11 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-27893702