Detalhe da pesquisa
1.
Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Haematologica
; 108(10): 2652-2663, 2023 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37021532
2.
Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice.
Haematologica
; 106(6): 1659-1670, 2021 06 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32354868
3.
Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients.
Am J Hematol
; 96(8): 989-999, 2021 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33984160
4.
Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells.
Gene Ther
; 27(9): 435-450, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32218505
5.
Advances in the gene therapy of monogenic blood cell diseases.
Clin Genet
; 97(1): 89-102, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31231794
6.
Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization.
Ann Hematol
; 99(5): 913-924, 2020 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-32065290
7.
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
em Inglês
| MEDLINE | ID: mdl-28801449
8.
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
em Inglês
| MEDLINE | ID: mdl-29503198
9.
Comparative Analysis between the In Vivo Biodistribution and Therapeutic Efficacy of Adipose-Derived Mesenchymal Stromal Cells Administered Intraperitoneally in Experimental Colitis.
Int J Mol Sci
; 19(7)2018 06 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-29937494
10.
Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
Mol Ther
; 24(7): 1187-98, 2016 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27138040
11.
Mutations in ERCC4, encoding the DNA-repair endonuclease XPF, cause Fanconi anemia.
Am J Hum Genet
; 92(5): 800-6, 2013 May 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-23623386
12.
Human Adipose-Derived Mesenchymal Stem Cells Modulate Experimental Autoimmune Arthritis by Modifying Early Adaptive T Cell Responses.
Stem Cells
; 33(12): 3493-503, 2015 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-26205964
13.
BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice.
J Pathol
; 236(2): 219-28, 2015 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-25664618
14.
Brief report: reduced expression of CD18 leads to the in vivo expansion of hematopoietic stem cells in mouse bone marrow.
Stem Cells
; 32(10): 2794-8, 2014 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-24906078
15.
Down-regulated expression of hsa-miR-181c in Fanconi anemia patients: implications in TNFα regulation and proliferation of hematopoietic progenitor cells.
Blood
; 119(13): 3042-9, 2012 Mar 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-22310912
16.
Brief report: impaired cell reprogramming in nonhomologous end joining deficient cells.
Stem Cells
; 31(8): 1726-30, 2013 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-23630174
17.
Origin, functional role, and clinical impact of Fanconi anemia FANCA mutations.
Blood
; 117(14): 3759-69, 2011 Apr 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-21273304
18.
Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases.
Hum Gene Ther
; 34(17-18): 763-775, 2023 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-37694572
19.
Improved efficacy of mesenchymal stromal cells stably expressing CXCR4 and IL-10 in a xenogeneic graft versus host disease mouse model.
Front Immunol
; 14: 1062086, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-36817457
20.
Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
Front Genome Ed
; 5: 1104666, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37188156