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1 . 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
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 .
Homozygous IL37 mutation associated with infantile inflammatory bowel disease
저자
by
Tingyan He
;
Yu Zhang
;
Ahmet Ozen
;
Helen C. Su
;
Colin L. Sweeney
, et al.
소스
Proceedings of the National Academy of Sciences of the United States of America
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7 .
Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells
저자
by
Naritaka Tamaoki
;
Stefan Siebert
;
Takuya Maeda
;
Ngoc-Han Ha
;
Meghan L. Good
, et al.
소스
Cell Reports Methods
. 3:100460
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7 .
Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells
저자
by
Matthew H. Porteus
;
Shengdar Q. Tsai
;
Siyuan Liu
;
Cicera R. Lazzarotto
;
Suk See De Ravin
, et al.
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Blood
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7 .
A Noncoding Germline Variant Creates a Gain-of-Function De Novo Enhancer That up-Regulates IL5 Transcription to Cause Familial Hypereosinophilia, Revealing a Previously Undescribed Mechanism for Human Disease
저자
by
Jung-Hyun Kim
;
Abbie Bowman
;
Senbagavalli Babu
;
Jan Cools
;
Sofie Demeyer
, et al.
소스
Blood
. 140:1399-1400
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7 .
MAGT1 messenger RNA-corrected autologous T and natural killer cells for potential cell therapy in X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection and neoplasia disease
저자
by
Linhong Li
;
Harry L. Malech
;
Kennichi C. Dowdell
;
Julie Brault
;
Ezekiel Bello
, et al.
소스
Cytotherapy
. 23:203-210
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7 .
CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes
저자
by
Ezekiel Bello
;
Marita Bosticardo
;
Ronald J. Meis
;
Sherry Koontz
;
Shengdar Q. Tsai
, et al.
소스
Blood
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7 .
Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human SCID-X1
저자
by
Narda L. Whiting-Theobald
;
Colin L. Sweeney
;
Tyra Estwick
;
Sandra Anaya-O'Brien
;
Siyuan Liu
, et al.
소스
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7 .
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
저자
by
Linhong Li
;
Ezekiel Bello
;
Harry L. Malech
;
Xiaolin Wu
;
Narda Theobald
, et al.
소스
Gene therapy
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