Detalhe da pesquisa
1.
Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.
Gene Ther
; 25(7): 454-472, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-30190607
2.
Gene therapy knockdown of VEGFR2 in retinal endothelial cells to treat retinopathy.
Angiogenesis
; 21(4): 751-764, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29730824
3.
Correction to: Gene therapy knockdown of VEGFR2 in retinal endothelial cells to treat retinopathy.
Angiogenesis
; 21(4): 765, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29943214
4.
Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in Hemophilia B mice.
Mol Ther
; 22(3): 567-574, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-23941813
5.
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.
Proc Natl Acad Sci U S A
; 106(4): 1205-10, 2009 Jan 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-19164558
6.
Targeted Knockdown of Overexpressed VEGFA or VEGF164 in Müller cells maintains retinal function by triggering different signaling mechanisms.
Sci Rep
; 8(1): 2003, 2018 01 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-29386650
7.
Toward Personalized Gene Therapy: Characterizing the Host Genetic Control of Lentiviral-Vector-Mediated Hepatic Gene Delivery.
Mol Ther Methods Clin Dev
; 5: 83-92, 2017 Jun 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-28480308
8.
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.
Hum Gene Ther
; 26(2): 69-81, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25419787
9.
Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder.
Mol Ther
; 6(6): 718-26, 2002 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-12498768
10.
Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides.
Mol Pharmacol
; 62(3): 545-53, 2002 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-12181431