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11 . Academic Journal
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
저자
by
Fumagalli, Francesca
;
Calbi, Valeria
;
Natali Sora, Maria Grazia
;
Sessa, Maria
;
Baldoli, Cristina
, et al.
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The Lancet
22-28 January 2022 399(10322):372-383
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17 . Academic Journal
From Your Nose to Your Toes: A Review of Severe Acute Respiratory Syndrome Coronavirus 2 Pandemic‒Associated Pernio
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Aiuti, Alessandro
;
Belot, Alexandre
;
Bolze, Alexandre
;
Bondarenko, Anastasiia
;
Sediva, Anna
, et al.
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Journal of Investigative Dermatology
December 2021 141(12):2791-2796
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17 . Academic Journal
Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations
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by
Boztug, Kaan
;
Brunner, Juergen
;
Demel, Ulrike F.
;
Förster-Waldl, Elisabeth
;
Gasteiger, Lukas M.
, et al.
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The Journal of Allergy and Clinical Immunology
November 2021 148(5):1332-1341
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17 . Academic Journal
Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency
저자
by
Zoccolillo, Matteo
;
Brigida, Immacolata
;
Barzaghi, Federica
;
Scala, Serena
;
Hernández, Raisa Jofra
, et al.
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Blood Advances
24 August 2021 5(16):3174-3187
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17 . Academic Journal
Treatment with rapamycin can restore regulatory T-cell function in IPEX patients
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by
Passerini, Laura
;
Barzaghi, Federica
;
Curto, Rosalia
;
Sartirana, Claudia
;
Barera, Graziano
, et al.
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The Journal of Allergy and Clinical Immunology
April 2020 145(4):1262-1271
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17 . Academic Journal
Reduced PD-1 expression on circulating follicular and conventional FOXP3+ Treg cells in children with new onset type 1 diabetes and autoantibody-positive at-risk children
저자
by
Vecchione, Andrea
;
Di Fonte, Roberta
;
Gerosa, Jolanda
;
Jofra, Tatiana
;
Cicalese, Maria Pia
, et al.
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Clinical Immunology
February 2020 211
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17 . Academic Journal
Gene therapy for rare haematological and neurometabolic paediatric diseases
저자
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Gallo, Vera
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Aiuti, Alessandro
.
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In
Global Pediatrics
September 2024 9
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14 . Academic Journal
3117 – HSCS IN Β-THALASSEMIA HAVE A REDUCED DORMANCY AND AN ERYTHROID BIASED LINEAGE COMMITMENT IN RESPONSE TO ALTERED TGFΒ SIGNALING IN THE BM MICROENVIRONMENT
저자
by
Lidonnici, Maria Rosa
;
Chianella, Giulia
;
Mende, Nicole
;
Bastos, Hugo
;
Barcella, Matteo
, et al.
소스
In
Experimental Hematology
August 2024 137 Supplement
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17 . Academic Journal
Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome
저자
by
Sereni, Lucia
;
Castiello, Maria Carmina
;
Di Silvestre, Dario
;
Della Valle, Patrizia
;
Brombin, Chiara
, et al.
소스
In
The Journal of Allergy and Clinical Immunology
September 2019 144(3):825-838
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17 . Academic Journal
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
저자
by
Ferrua, Francesca
;
Cicalese, Maria Pia
;
Galimberti, Stefania
;
Giannelli, Stefania
;
Dionisio, Francesca
, et al.
소스
In
The Lancet Haematology
May 2019 6(5):e239-e253
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