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1 . Academic Journal
A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome
저자
by
David H. McDermott
;
Daniel Velez
;
Elena Cho
;
Edward W. Cowen
;
John J. DiGiovanna
, et al.
소스
The Journal of Clinical Investigation, Vol 133, Iss 19 (2023)
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7 . Academic Journal
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency
저자
by
Suk See De Ravin
;
Siyuan Liu
;
Colin L. Sweeney
;
Julie Brault
;
Narda Whiting-Theobald
, et al.
소스
Nature Communications, Vol 13, Iss 1, Pp 1-15 (2022)
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7 . Academic Journal
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells
저자
by
Julie Brault
;
Taylor Liu
;
Siyuan Liu
;
Amanda Lawson
;
Uimook Choi
, et al.
소스
Frontiers in Immunology, Vol 13 (2023)
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7 . Academic Journal
Homozygous variant p. Arg90His in NCF1 is associated with early-onset Interferonopathy: a case report
저자
by
Oskar Schnappauf
;
Liane Heale
;
Dilan Dissanayake
;
Wanxia L. Tsai
;
Massimo Gadina
, et al.
소스
Pediatric Rheumatology Online Journal, Vol 19, Iss 1, Pp 1-8 (2021)
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7 . Academic Journal
Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models
저자
by
Naoya Uchida
;
Linhong Li
;
Tina Nassehi
;
Claire M. Drysdale
;
Morgan Yapundich
, et al.
소스
Cell Reports Medicine, Vol 2, Iss 4, Pp 100247- (2021)
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7 . Academic Journal
Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease
저자
by
Randall K. Merling
;
Douglas B. Kuhns
;
Colin L. Sweeney
;
Xiaolin Wu
;
Sandra Burkett
, et al.
소스
Blood Advances, Vol 1, Iss 4, Pp 270-278 (2017)
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7 . Academic Journal
Granulibacter bethesdensis, a Pathogen from Patients with Chronic Granulomatous Disease, Produces a Penta-Acylated Hypostimulatory Glycero-D-talo-oct-2-ulosonic Acid–Lipid A Glycolipid (Ko-Lipid A)
저자
by
Artur Muszyński
;
Kol A. Zarember
;
Christian Heiss
;
Joseph Shiloach
;
Lars J. Berg
, et al.
소스
International Journal of Molecular Sciences, Vol 22, Iss 7, p 3303 (2021)
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7 . Academic Journal
Recurrent Granulibacter bethesdensis Infections and Chronic Granulomatous Disease
저자
by
David E. Greenberg
;
Adam R. Shoffner
;
Adrian M. Zelazny
;
Michael E. Fenster
;
Kol A. Zarember
, et al.
소스
Emerging Infectious Diseases, Vol 16, Iss 9, Pp 1341-1348 (2010)
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7 .
Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease
저자
by
Alexander H. Yang
;
Brigit Sullivan
;
Christa S. Zerbe
;
Suk See De Ravin
;
Andrew M. Blakely
, et al.
소스
The Journal of Allergy and Clinical Immunology: In Practice
. 11:1401-1416
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7 .
Gene therapy for inborn errors of immunity: Base editing comes into play
저자
by
Harry L. Malech
;
Luigi D. Notarangelo
.
소스
Cell
. 186:1302-1304
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