Detalhe da pesquisa
1.
Initiation of Otx2 expression in the developing mouse retina requires a unique enhancer and either Ascl1 or Neurog2 activity.
Development
; 148(12)2021 06 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-34143204
2.
Binge ethanol exposure in advanced age elevates neuroinflammation and early indicators of neurodegeneration and cognitive impairment in female mice.
Brain Behav Immun
; 116: 303-316, 2024 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-38151165
3.
An enhancer located in a Pde6c intron drives transient expression in the cone photoreceptors of developing mouse and human retinas.
Dev Biol
; 488: 131-150, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35644251
4.
Transcriptional profiling of murine retinas undergoing semi-synchronous cone photoreceptor differentiation.
Dev Biol
; 453(2): 155-167, 2019 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-31163126
5.
Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair.
Stem Cells
; 37(2): 284-294, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30372555
6.
Corrigendum to "Binge ethanol exposure in advanced age elevates neuroinflammation and early indicators of neurodegeneration and cognitive impairment in female mice" [Brain Behav. Immun. 116 (2024) 303-316].
Brain Behav Immun
; 2024 May 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-38702200
7.
Characterization of Gene Alterations following Editing of the ß-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells.
Mol Ther
; 26(2): 468-479, 2018 02 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29221806
8.
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
Blood
; 125(17): 2597-604, 2015 Apr 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-25733580
9.
Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.
Cytotherapy
; 19(9): 1096-1112, 2017 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-28733131
10.
Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy.
Stem Cells
; 33(5): 1532-42, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25588820
11.
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Mol Ther
; 22(3): 607-622, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-24256635
12.
Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction.
Blood
; 120(18): 3677-87, 2012 Nov 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-22833548
13.
Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells.
Mol Ther
; 21(5): 1044-54, 2013 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-23380815
14.
In Situ Spatial Reconstruction of Distinct Normal and Pathological Cell Populations Within the Human Adrenal Gland.
J Endocr Soc
; 7(12): bvad131, 2023 Nov 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-37953901
15.
Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID.
Mol Ther Methods Clin Dev
; 20: 765-778, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33738330
16.
Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice.
Mol Ther Methods Clin Dev
; 16: 78-93, 2020 Mar 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31871959
17.
Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
Hum Gene Ther
; 29(10): 1153-1166, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-30198339
18.
Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome.
Cell Rep
; 23(9): 2606-2616, 2018 05 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-29847792
19.
The human ankyrin 1 promoter insulator sustains gene expression in a ß-globin lentiviral vector in hematopoietic stem cells.
Mol Ther Methods Clin Dev
; 2: 15012, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26029723
20.
Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells.
Exp Hematol
; 43(5): 346-351, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25681747