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1 .
Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease
저자
by
Xiaoyan Wang
;
Fabrizia Urbinati
;
Filippo Marchioni
;
Aaron R. Cooper
;
Barbara Nowicki
, et al.
소스
Cytotherapy
. 19:1096-1112
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7 .
Molecular, phenotypic, and sample-associated data to describe pluripotent stem cell lines and derivatives
저자
by
Robin Schroll
;
Nathan Salomonis
;
Stacy Bush
;
Mehdi Keddache
;
Bruce J. Aronow
, et al.
소스
Scientific Data
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7 .
Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype
저자
by
Stella M. Davies
;
Archana Shreshta
;
Johannes C.M. Van der Loo
;
Jennifer Knight-Madden
;
Theodosia A. Kalfa
, et al.
소스
Blood
. 132:1021-1021
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7 .
Overcoming reprogramming resistance of Fanconi anemia cells
저자
by
Wendy B. London
;
Chad E. Harris
;
Rutesh Vyas
;
Thorsten M. Schlaeger
;
Alex Devine
, et al.
소스
Blood
. 119:5449-5457
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7 .
FAAP20: a novel ubiquitin-binding FA nuclear core-complex protein required for functional integrity of the FA-BRCA DNA repair pathway
저자
by
Arun Pradhan
;
Kebola Wahengbam
;
Chang-hu Du
;
Abdullah Mahmood Ali
;
Thiyam Ramsingh Singh
, et al.
소스
Blood
. 119:3285-3294
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7 .
Interlaboratory assessment of a novel colony-forming unit assay: a multicenter study by the cellular team of Biomedical Excellence for Safer Transfusion (BEST) collaborative
저자
by
Maria Nawrot
;
Toni Temples
;
Ning Yuan
;
Elke Grassman
;
Zbigniew M. Szczepiorkowski
, et al.
소스
Transfusion
. 51:2001-2005
Full Text (Wiley)
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7 .
Correct mRNA Processing at a Mutant TT Splice Donor in FANCC Ameliorates the Clinical Phenotype in Patients and Is Enhanced by Delivery of Suppressor U1 snRNAs
저자
by
Stephan Theiss
;
Kornelia Neveling
;
Helmut Hanenberg
;
Linda Hartmann
;
Stefan Burdach
, et al.
소스
The American Journal of Human Genetics
. 87(4):480-493
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7 .
Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors
저자
by
Ute Modlich
;
Anjali Mishra
;
Christopher Baum
;
Daniela Zychlinski
;
Elke Grassman
, et al.
소스
Molecular Therapy
. 16(4):718-725
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7 .
Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency
저자
by
Meera Ulaganathan
;
Steven J. Howe
;
Elke Grassman
;
Axel Schambach
;
H. Bobby Gaspar
, et al.
소스
Molecular Therapy
. 16:590-598
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7 .
Cell and Virus Genetics at the Roots of Gene Therapy, Retrovirology, and Hematopoietic Stem Cell Biology: Wolfram Ostertag (1937–2010)
저자
by
Juergen Bode
;
Christopher Baum
;
Schröder T
;
Hannes Klump
;
Olga S. Kustikova
, et al.
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Scopus-Elsevier
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