Detalhe da pesquisa
1.
Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing.
Cell
; 186(7): 1398-1416.e23, 2023 03 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-36944331
2.
Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades.
Immunol Rev
; 322(1): 148-156, 2024 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-38033164
3.
Diverse Approaches to Gene Therapy of Sickle Cell Disease.
Annu Rev Med
; 74: 473-487, 2023 01 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-36067800
4.
Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.
Blood
; 141(9): 1007-1022, 2023 03 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36332160
5.
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study.
J Allergy Clin Immunol
; 153(1): 287-296, 2024 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-37793572
6.
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study.
J Allergy Clin Immunol
; 153(5): 1423-1431.e2, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38290608
7.
Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia.
Clin Immunol
; 261: 109942, 2024 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-38367737
8.
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
9.
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.
Blood
; 140(7): 685-705, 2022 08 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-35671392
10.
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials.
Mol Ther
; 31(3): 801-809, 2023 03 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36518078
11.
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions.
J Allergy Clin Immunol
; 151(2): 539-546, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36456361
12.
Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation.
J Allergy Clin Immunol
; 151(1): 260-271, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35987350
13.
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions.
J Allergy Clin Immunol
; 151(2): 547-555.e5, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36456360
14.
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease.
J Allergy Clin Immunol
; 152(6): 1619-1633.e11, 2023 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-37659505
15.
Successes and challenges in clinical gene therapy.
Gene Ther
; 30(10-11): 738-746, 2023 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-37935854
16.
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential.
Gene Ther
; 30(3-4): 369-376, 2023 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-36216880
17.
Biodistribution of lentiviral transduced adipose-derived stem cells for "ex-vivo" regional gene therapy for bone repair.
Gene Ther
; 30(12): 826-834, 2023 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-37568039
18.
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
19.
Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy.
J Clin Immunol
; 42(5): 1026-1035, 2022 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-35445907
20.
Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.
Blood
; 135(23): 2094-2105, 2020 06 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32268350