Detalhe da pesquisa
1.
Comparative analysis of lentiviral gene transfer approaches designed to promote fetal hemoglobin production for the treatment of ß-hemoglobinopathies.
Blood Cells Mol Dis
; 84: 102456, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32498026
2.
Generation of a heterozygous and a homozygous CSF1R knockout line from iPSC using CRISPR/Cas9.
Stem Cell Res
; 69: 103066, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-36947995
3.
Automated Good Manufacturing Practice-Compatible CRISPR-Cas9 Editing of Hematopoietic Stem and Progenitor Cells for Clinical Treatment of ß-Hemoglobinopathies.
CRISPR J
; 6(1): 5-16, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36662546
4.
A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy.
CRISPR J
; 5(1): 66-79, 2022 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34882002
5.
Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing.
Genes (Basel)
; 13(12)2022 12 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-36553615
6.
Universal Gene Correction Approaches for ß-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates.
CRISPR J
; 4(2): 207-222, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33876951
7.
Autoantibodies against the immunodominant sCha epitope discriminate the risk of sudden death in chronic Chagas cardiomyopathy.
Ann N Y Acad Sci
; 1497(1): 27-38, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-33682151
8.
Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches.
Blood Rev
; 40: 100641, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31761379
9.
Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin.
Sci Rep
; 10(1): 10133, 2020 06 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-32576837
10.
CRISPR/Cas9 technology: towards a new generation of improved CAR-T cells for anticancer therapies.
Brief Funct Genomics
; 19(3): 191-200, 2020 05 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-31844895
11.
CRISPR/Cas9-modified hematopoietic stem cells-present and future perspectives for stem cell transplantation.
Bone Marrow Transplant
; 54(12): 1940-1950, 2019 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-30903024
12.
Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors.
Mol Cell Pediatr
; 5(1): 9, 2018 Nov 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-30430274