Detalhe da pesquisa
1.
GSE4-loaded nanoparticles a potential therapy for lung fibrosis that enhances pneumocyte growth, reduces apoptosis and DNA damage.
FASEB J
; 35(3): e21422, 2021 03.
Artigo
Inglês
| MEDLINE | ID: mdl-33638895
2.
Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells.
Gene Ther
; 27(9): 435-450, 2020 09.
Artigo
Inglês
| MEDLINE | ID: mdl-32218505
3.
Advances in the gene therapy of monogenic blood cell diseases.
Clin Genet
; 97(1): 89-102, 2020 01.
Artigo
Inglês
| MEDLINE | ID: mdl-31231794
4.
Mutations in XLF/NHEJ1/Cernunnos gene results in downregulation of telomerase genes expression and telomere shortening.
Hum Mol Genet
; 26(10): 1900-1914, 2017 05 15.
Artigo
Inglês
| MEDLINE | ID: mdl-28369633
5.
Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients.
Blood
; 130(13): 1535-1542, 2017 09 28.
Artigo
Inglês
| MEDLINE | ID: mdl-28801449
6.
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.
Mol Ther
; 26(4): 1137-1153, 2018 04 04.
Artigo
Inglês
| MEDLINE | ID: mdl-29503198
7.
Conversion of human fibroblasts into monocyte-like progenitor cells.
Stem Cells
; 32(11): 2923-2938, 2014 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-25175072
8.
Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.
Nature
; 460(7251): 53-9, 2009 Jul 02.
Artigo
Inglês
| MEDLINE | ID: mdl-19483674
9.
Brief report: impaired cell reprogramming in nonhomologous end joining deficient cells.
Stem Cells
; 31(8): 1726-30, 2013 Aug.
Artigo
Inglês
| MEDLINE | ID: mdl-23630174
10.
Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1.
Stem Cell Res Ther
; 12(1): 92, 2021 01 29.
Artigo
Inglês
| MEDLINE | ID: mdl-33514435
11.
Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors.
Blood
; 112(8): 3138-47, 2008 Oct 15.
Artigo
Inglês
| MEDLINE | ID: mdl-18684860
12.
In vivo proliferation advantage of genetically corrected hematopoietic stem cells in a mouse model of Fanconi anemia FA-D1.
Blood
; 112(13): 4853-61, 2008 Dec 15.
Artigo
Inglês
| MEDLINE | ID: mdl-18812474
13.
Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients.
Mol Ther
; 17(6): 1083-92, 2009 Jun.
Artigo
Inglês
| MEDLINE | ID: mdl-19277017
14.
Immunomagnetic enrichment of human and mouse hematopoietic stem cells for gene therapy applications.
Methods Mol Biol
; 506: 1-11, 2009.
Artigo
Inglês
| MEDLINE | ID: mdl-19110615
15.
GSE4 peptide suppresses oxidative and telomere deficiencies in ataxia telangiectasia patient cells.
Cell Death Differ
; 26(10): 1998-2014, 2019 Oct.
Artigo
Inglês
| MEDLINE | ID: mdl-30670828
16.
Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Nat Med
; 25(9): 1396-1401, 2019 09.
Artigo
Inglês
| MEDLINE | ID: mdl-31501599
17.
Regulatory elements of the vav gene drive transgene expression in hematopoietic stem cells from adult mice.
Exp Hematol
; 32(4): 360-4, 2004 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-15050746
18.
Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs.
Curr Gene Ther
; 15(6): 550-62, 2015.
Artigo
Inglês
| MEDLINE | ID: mdl-26415575
19.
Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells.
Stem Cell Reports
; 5(6): 1053-1066, 2015 Dec 08.
Artigo
Inglês
| MEDLINE | ID: mdl-26549847
20.
Genetic modification of hematopoietic stem cells: recent advances in the gene therapy of inherited diseases.
Arch Med Res
; 34(6): 589-99, 2003.
Artigo
Inglês
| MEDLINE | ID: mdl-14734099