Detalhe da pesquisa
1.
An optimized SpCas9 high-fidelity variant for direct protein delivery.
Mol Ther
; 31(7): 2257-2265, 2023 07 05.
Artigo
Inglês
| MEDLINE | ID: mdl-36905119
2.
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
Mol Ther
; 30(1): 145-163, 2022 01 05.
Artigo
Inglês
| MEDLINE | ID: mdl-34418541
3.
ABCG2 Is Overexpressed on Red Blood Cells in Ph-Negative Myeloproliferative Neoplasms and Potentiates Ruxolitinib-Induced Apoptosis.
Int J Mol Sci
; 22(7)2021 Mar 29.
Artigo
Inglês
| MEDLINE | ID: mdl-33805426
4.
Impact of hydroxycarbamide and interferon-α on red cell adhesion and membrane protein expression in polycythemia vera.
Haematologica
; 103(6): 972-981, 2018 06.
Artigo
Inglês
| MEDLINE | ID: mdl-29599206
5.
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
Mol Ther Nucleic Acids
; 32: 229-246, 2023 Jun 13.
Artigo
Inglês
| MEDLINE | ID: mdl-37090420
6.
Enhanced calreticulin expression in red cells of polycythemia vera patients harboring the JAK2V617F mutation.
Haematologica
; 102(7): e241-e244, 2017 07.
Artigo
Inglês
| MEDLINE | ID: mdl-28385780
7.
CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy.
Stem Cells Transl Med
; 11(10): 1003-1009, 2022 Oct 21.
Artigo
Inglês
| MEDLINE | ID: mdl-36048170
8.
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.
Nat Commun
; 13(1): 6618, 2022 11 04.
Artigo
Inglês
| MEDLINE | ID: mdl-36333351
9.
Genome editing approaches to ß-hemoglobinopathies.
Prog Mol Biol Transl Sci
; 182: 153-183, 2021.
Artigo
Inglês
| MEDLINE | ID: mdl-34175041
10.
Base and Prime Editing Technologies for Blood Disorders.
Front Genome Ed
; 3: 618406, 2021.
Artigo
Inglês
| MEDLINE | ID: mdl-34713251
11.
Endothelial Cells Harbouring the JAK2V617F Mutation Display Pro-Adherent and Pro-Thrombotic Features.
Thromb Haemost
; 118(9): 1586-1599, 2018 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-30103245
12.
Abnormal adhesion of red blood cells in polycythemia vera: a prothrombotic effect?
Thromb Res
; 133 Suppl 2: S107-11, 2014 May.
Artigo
Inglês
| MEDLINE | ID: mdl-24862129