Detalhe da pesquisa
1.
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.
Mol Ther
; 2024 Mar 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-38549377
2.
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.
Mol Ther
; 31(3): 657-675, 2023 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36457248
3.
The transformative potential of HSC gene therapy as a genetic medicine.
Gene Ther
; 30(3-4): 197-215, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-34040164
4.
Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.
J Clin Immunol
; 43(2): 440-451, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36329240
5.
Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience.
J Clin Immunol
; 42(1): 94-107, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34654999
6.
Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood.
J Clin Immunol
; 42(6): 1230-1243, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35579633
7.
Clinical and immunological features in a cohort of patients with partial DiGeorge syndrome followed at a single center.
Blood
; 133(24): 2586-2596, 2019 06 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31015189
8.
Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.
Biol Blood Marrow Transplant
; 26(10): 1819-1827, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32653625
9.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
N Engl J Med
; 377(17): 1630-1638, 2017 10 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28976817
10.
Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.
J Allergy Clin Immunol
; 143(3): 852-863, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30194989
11.
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.
Blood
; 130(11): 1327-1335, 2017 09 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-28716862
12.
Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease.
J Allergy Clin Immunol
; 142(1): 235-245.e6, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29705247
13.
T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.
J Allergy Clin Immunol
; 142(3): 904-913.e3, 2018 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-29355678
14.
Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders.
J Allergy Clin Immunol
; 141(1): 322-328.e10, 2018 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28392333
15.
Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.
Mol Ther
; 25(8): 1805-1814, 2017 08 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28434866
16.
Mutations in linker for activation of T cells (LAT) lead to a novel form of severe combined immunodeficiency.
J Allergy Clin Immunol
; 139(2): 634-642.e5, 2017 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-27522155
17.
Thymus transplantation for complete DiGeorge syndrome: European experience.
J Allergy Clin Immunol
; 140(6): 1660-1670.e16, 2017 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-28400115
18.
A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis.
J Allergy Clin Immunol
; 139(4): 1302-1310.e4, 2017 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-27658761
19.
How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).
J Clin Immunol
; 37(4): 351-356, 2017 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-28194615
20.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
N Engl J Med
; 371(15): 1407-17, 2014 Oct 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-25295500