Detalhe da pesquisa
1.
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
N Engl J Med
; 384(21): 2002-2013, 2021 05 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-33974366
2.
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
3.
Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.
Blood
; 129(19): 2624-2635, 2017 05 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-28351939
4.
Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.
J Clin Immunol
; 37(7): 626-637, 2017 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-28842866
5.
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
6.
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
7.
Letter to the Editor. Differences between lentiviral vector-mediated ex vivo hematopoietic stem cell and adeno-associated virus gene therapies for neurological disorders.
J Neurosurg
; 136(5): 1501, 2021 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-34891135