Detalhe da pesquisa
1.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
N Engl J Med
; 385(21): 1929-1940, 2021 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-34788506
2.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35065785
3.
Strategies for single base gene editing in an immortalized human cell line by CRISPR/Cas9 technology.
3 Biotech
; 14(2): 45, 2024 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-38261961
4.
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Sci Transl Med
; 16(745): eadi8214, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38691622
5.
Pro64His (rs4644) Polymorphism Within Galectin-3 Is a Risk Factor of Differentiated Thyroid Carcinoma and Affects the Transcriptome of Thyrocytes Engineered via CRISPR/Cas9 System.
Thyroid
; 31(7): 1056-1066, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33308024
6.
Variation rs2235503 C > A Within the Promoter of MSLN Affects Transcriptional Rate of Mesothelin and Plasmatic Levels of the Soluble Mesothelin-Related Peptide.
Front Genet
; 11: 975, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-33014022