Detalhe da pesquisa
1.
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Blood
; 142(15): 1281-1296, 2023 10 12.
Artigo
Inglês
| MEDLINE | ID: mdl-37478401
2.
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
N Engl J Med
; 384(3): 205-215, 2021 01 21.
Artigo
Inglês
| MEDLINE | ID: mdl-33283990
3.
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
N Engl J Med
; 381(17): 1644-1652, 2019 10 24.
Artigo
Inglês
| MEDLINE | ID: mdl-31597037
4.
Mechanisms underlying genetic susceptibility to multisystem inflammatory syndrome in children (MIS-C).
J Allergy Clin Immunol
; 148(3): 732-738.e1, 2021 09.
Artigo
Inglês
| MEDLINE | ID: mdl-34224783
5.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
N Engl J Med
; 377(17): 1630-1638, 2017 10 26.
Artigo
Inglês
| MEDLINE | ID: mdl-28976817
6.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
N Engl J Med
; 371(15): 1407-17, 2014 Oct 09.
Artigo
Inglês
| MEDLINE | ID: mdl-25295500
7.
Prostaglandin-modulated umbilical cord blood hematopoietic stem cell transplantation.
Blood
; 122(17): 3074-81, 2013 Oct 24.
Artigo
Inglês
| MEDLINE | ID: mdl-23996087
8.
Cellular Therapies Clinical Research Roadmap: lessons learned on how to move a cellular therapy into a clinical trial.
Cytotherapy
; 17(4): 339-43, 2015 Apr.
Artigo
Inglês
| MEDLINE | ID: mdl-25484311
9.
Viral induction and targeted inhibition of galectin-1 in EBV+ posttransplant lymphoproliferative disorders.
Blood
; 117(16): 4315-22, 2011 Apr 21.
Artigo
Inglês
| MEDLINE | ID: mdl-21300977
10.
Modulation of Radiation Biomarkers in a Randomized Phase II Study of 131I-MIBG With or Without Radiation Sensitizers for Relapsed or Refractory Neuroblastoma.
Int J Radiat Oncol Biol Phys
; 115(5): 1115-1128, 2023 04 01.
Artigo
Inglês
| MEDLINE | ID: mdl-36526235
11.
Human genetic diversity alters off-target outcomes of therapeutic gene editing.
Nat Genet
; 55(1): 34-43, 2023 Jan.
Artigo
Inglês
| MEDLINE | ID: mdl-36522432
12.
Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease.
Nat Med
; 29(12): 3175-3183, 2023 Dec.
Artigo
Inglês
| MEDLINE | ID: mdl-37973947
13.
Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling.
Nat Commun
; 14(1): 5850, 2023 09 20.
Artigo
Inglês
| MEDLINE | ID: mdl-37730674
14.
Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety.
Sci Adv
; 9(33): eadg6470, 2023 08 18.
Artigo
Inglês
| MEDLINE | ID: mdl-37595035
15.
Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells.
bioRxiv
; 2023 May 27.
Artigo
Inglês
| MEDLINE | ID: mdl-37292647
16.
Cultivated Autologous Limbal Epithelial Cell Transplantation: New Frontier in the Treatment of Limbal Stem Cell Deficiency.
Am J Ophthalmol
; 239: 244-268, 2022 07.
Artigo
Inglês
| MEDLINE | ID: mdl-35314191
17.
Metformin for treatment of cytopenias in children and young adults with Fanconi anemia.
Blood Adv
; 6(12): 3803-3811, 2022 06 28.
Artigo
Inglês
| MEDLINE | ID: mdl-35500223
18.
Enhanced induction of HIV-specific cytotoxic T lymphocytes by dendritic cell-targeted delivery of SOCS-1 siRNA.
Mol Ther
; 18(11): 2028-37, 2010 Nov.
Artigo
Inglês
| MEDLINE | ID: mdl-20648001
19.
Dissecting ELANE neutropenia pathogenicity by human HSC gene editing.
Cell Stem Cell
; 28(5): 833-845.e5, 2021 05 06.
Artigo
Inglês
| MEDLINE | ID: mdl-33513358
20.
Cell therapy product administration and safety: data capture and analysis from the Production Assistance for Cellular Therapies (PACT) program.
Transfusion
; 55(3): 674-9, 2015 Mar.
Artigo
Inglês
| MEDLINE | ID: mdl-25315143