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1 . Academic Journal
Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis
저자
by
Quaranta, Pamela
;
Basso-Ricci, Luca
;
Jofra Hernandez, Raisa
;
Pacini, Guido
;
Naldini, Matteo Maria
, et al.
소스
In
Blood
9 May 2024 143(19):1937-1952
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7 . Academic Journal
Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study
저자
by
Aiuti, Alessandro
;
Maschan, Alexei
;
Aljurf, Mahmoud
;
Gedde-Dahl, Tobias
;
Gurman, Gunhan
, et al.
소스
In
Blood
6 October 2022 140(14):1635-1649
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7 . Academic Journal
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis
저자
by
Albert, Michael H.
;
Slatter, Mary A.
;
Gennery, Andrew R.
;
Güngör, Tayfun
;
Bakunina, Katerina
, et al.
소스
In
Blood
31 March 2022 139(13):2066-2079
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7 . 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.
소스
In
The Lancet
22-28 January 2022 399(10322):372-383
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7 . 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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7 . Academic Journal
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future
저자
by
Taylor, Madeleine
;
Khan, Shaukat
;
Stapleton, Molly
;
Wang, Jianmin
;
Chen, Jing
, et al.
소스
In
Biology of Blood and Marrow Transplantation
July 2019 25(7):e226-e246
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7 . 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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7 . Academic Journal
Current practices for nutritional evaluation and care during the treatment of pediatric oncology patients: a survey among AIEOP centers.
저자
by
Venturelli, Francesco
;
Leardini, Davide
;
Baccelli, Francesco
;
Gottardi, Francesca
;
Barat, Veronica
, et al.
소스
European Journal of Pediatrics
. Mar2024, Vol. 183 Issue 3, p1137-1144. 8p.
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7 . Academic Journal
Design of a multi-center randomized active controlled phase 3 clinical trial (HURCULES) evaluating the safety and efficacy of OTL-203 in patients with MPS IH versus standard of care with allogeneic hematopoietic stem cell transplantation
저자
by
Orchard, Paul J.
;
Wynn, Robert F.
;
Kharbanda, Sandhya
;
Ahrens-Nicklas, Rebecca
;
van Hasselt, Peter M.
, et al.
소스
In
Molecular Genetics and Metabolism
February 2024 141(2)
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7 . Academic Journal
P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation
저자
by
Harmatz, Paul
;
Wynn, Robert
;
Gupta, Ashish
;
Kharbanda, Sandhya
;
Lindemans, Caroline
, et al.
소스
In
Genetics in Medicine Open
2024 2 Supplement 1
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