Detalhe da pesquisa
1.
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
Blood
; 138(15): 1304-1316, 2021 10 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-33974038
2.
Ribosomal and hematopoietic defects in induced pluripotent stem cells derived from Diamond Blackfan anemia patients.
Blood
; 122(6): 912-21, 2013 Aug 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-23744582
3.
Single nucleotide polymorphism array analysis of bone marrow failure patients reveals characteristic patterns of genetic changes.
Br J Haematol
; 164(1): 73-82, 2014 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-24116929
4.
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.
Blood
; 119(13): 3038-41, 2012 Mar 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-22271447
5.
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
Blood
; 120(18): 3635-46, 2012 Nov 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-22968453
6.
Somatic HLA Mutations Expose the Role of Class I-Mediated Autoimmunity in Aplastic Anemia and its Clonal Complications.
Blood Adv
; 1(22): 1900-1910, 2017 Oct 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-28971166
7.
Impaired Telomere Maintenance and Decreased Canonical WNT Signaling but Normal Ribosome Biogenesis in Induced Pluripotent Stem Cells from X-Linked Dyskeratosis Congenita Patients.
PLoS One
; 10(5): e0127414, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-25992652
8.
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes.
Mol Ther Methods Clin Dev
; 2: 15029, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-26445723
9.
Emergence of clonal hematopoiesis in the majority of patients with acquired aplastic anemia.
Cancer Genet
; 208(4): 115-28, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25800665
10.
Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans.
Hum Gene Ther
; 24(4): 431-42, 2013 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-23461589
11.
Overcoming preexisting humoral immunity to AAV using capsid decoys.
Sci Transl Med
; 5(194): 194ra92, 2013 Jul 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-23863832