Gene therapy goes the distance in Wiskott-Aldrich syndrome

被引：1

作者：

Biffi, Alessandra ^{[1
,2
]}

机构：

[1] Padua Univ Hosp, Pediat Hematol Oncol & Stem Cell Transplant Div, Padua, Italy

[2] Dana Farber Boston Childrens Canc & Blood Disorde, Gene Therapy Program, Boston, MA 02115 USA

来源：

关键词：

EFFICACY;

D O I：

10.1038/s41591-021-01653-7

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Long-term follow-up data reinforce the curative potential of hematopoietic stem-cell gene therapy for this rare primary immunodeficiency disorder.

引用

页码：24 / 25

页数：2

共 50 条

[41] THE WISKOTT-ALDRICH SYNDROME
PEACOCKE, M
SIMINOVITCH, KA
[J]. SEMINARS IN DERMATOLOGY, 1993, 12 (03): : 247 - 254
[42] Wiskott-Aldrich Syndrome
Kariya, Shin
Nishizaki, Kazunori
Paparella, Michael M.
Cureoglu, Sebahattin
[J]. OTOLOGY & NEUROTOLOGY, 2013, 34 (03) : E16 - E17
[43] WISKOTT-ALDRICH SYNDROME
ERTTMANN, R
THONE, I
LANDBECK, G
[J]. MONATSSCHRIFT KINDERHEILKUNDE, 1983, 131 (08) : 524 - 527
[44] The Wiskott-Aldrich syndrome
Ochs, HD
[J]. CLINICAL REVIEWS IN ALLERGY & IMMUNOLOGY, 2001, 20 (01) : 61 - 86
[45] MUTATIONS WITHIN THE WISKOTT-ALDRICH SYNDROME GENE
ZHU, QL
ZHANG, M
BLAESE, M
JUNKER, A
OCHS, HD
[J]. PEDIATRIC RESEARCH, 1995, 37 (04) : A12 - A12
[46] The Wiskott-Aldrich syndrome
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Thrasher, AJ
[J]. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2006, 117 (04) : 725 - 738
[47] The Wiskott-Aldrich syndrome
J. S. Orange
K. D. Stone
S. E. Turvey
K. Krzewski
[J]. Cellular and Molecular Life Sciences CMLS, 2004, 61 : 2361 - 2385
[48] Wiskott-Aldrich Syndrome (WAS)
Schuster, V
Böhler, T
Klein, C
[J]. ALLERGOLOGIE, 2004, 27 (04) : 184 - 188
[49] Wiskott-Aldrich syndrome
Notarangelo, Luigi D.
Miao, Carol H.
Ochs, Hans D.
[J]. CURRENT OPINION IN HEMATOLOGY, 2008, 15 (01) : 30 - 36
[50] The Wiskott-Aldrich syndrome
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[J]. SPRINGER SEMINARS IN IMMUNOPATHOLOGY, 1998, 19 (04): : 435 - 458