Outcomes of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

被引：0

作者：

Labrosse, Roxane ^{[1
]}

Chu, Julia ^{[2
]}

Armant, Myriam ^{[3
]}

Van der Spek, Jet ^{[4
]}

Miggelbrink, Alexandra ^{[1
]}

Fong, Johnson ^{[4
]}

Everett, John ^{[5
]}

Raymond, Hayley ^{[5
]}

Kessler, Lyanna ^{[5
]}

Dansereau, Colleen ^{[6
]}

Mackinnon, Brenda ^{[7
]}

Koo, Stephanie ^{[7
]}

Morris, Emily ^{[7
]}

London, Wendy ^{[8
]}

Ozen, Ahmet ^{[9
]}

Baris, Safa ^{[9
]}

Despotovic, Jenny ^{[10
]}

Forbes, Lisa ^{[11
]}

Saitoh, Akihiko ^{[12
]}

Takachi, Takayuki ^{[13
]}

King, Alejandra ^{[14
]}

Mai Anh Thi Nguyen ^{[15
]}

Uyen Vy Vu Do ^{[15
]}

Bushman, Frederic ^{[15
,16
]}

Galy, Anne ^{[17
]}

Notarangelo, Luigi ^{[18
]}

Williams, David ^{[4
]}

Pai, Sung-Yun ^{[4
]}

机构：

[1] Boston Childrens Hosp, Div Pediat Hematol Oncol, Boston, MA USA

[2] Boston Childrens Hosp, Dana Farber Canc Inst, Boston, MA USA

[3] Boston Childrens Hosp, Pediat, Boston, MA USA

[4] Boston Childrens Hosp, Div Hematol Oncol, Boston, MA USA

[5] Univ Penn, Perelman Sch Med, Dept Microbiol, Philadelphia, PA 19104 USA

[6] Dana Farber Boston Childrens Canc & Blood Disorde, Clin Operat Gene Therapy Program, Boston, MA USA

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

[8] Dana Farber Childrens Hosp Canc Ctr, Div Pediat Hematol Oncol, Pediat, Boston, MA USA

[9] Marmara Univ, Istanbul, Turkey

[10] Baylor Coll Med, Texas Childrens Hosp, Houston, TX 77030 USA

[11] Baylor Coll Med, Texas Childrens Hosp, Pediat, Houston, TX 77030 USA

[12] Niigata Univ, Grad Sch Med & Dent Sci, Niigata, Japan

[13] Shizuoka Childrens Hosp, Dept Hematol & Oncol, Shizuoka, Japan

[14] Hosp Ninos Luis Calvo Mackenna, Immunol Unit, Santiago, Chile

[15] City Childrens Hosp, Dept Hematol Oncol, Ho Chi Minh City, Vietnam

[16] Univ Penn, Perelman Sch Med, Dept Microbiol, Microbiol, Philadelphia, PA 19104 USA

[17] Genethon, Evry, France

[18] NIAID, Lab Clin Immunol & Microbiol, NIH, 9000 Rockville Pike, Bethesda, MD 20892 USA

来源：

JOURNAL OF CLINICAL IMMUNOLOGY | 2020年 / 40卷 / SUPPL 1期

关键词：

D O I：

暂无

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

804727

引用

页码：S28 / S29

页数：2

共 50 条

[1] Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome
Labrosse, Roxane
Chu, Julia I.
Armant, Myriam A.
Everett, John K.
Pellin, Danilo
Kareddy, Niharika
Frelinger, Andrew L.
Henderson, Lauren A.
O'Connell, Amy E.
Biswas, Amlan
Coenen-van der Spek, Jet
Miggelbrink, Alexandra
Fiorini, Claudia
Adhikari, Hriju
Berry, Charles C.
Cantu, Vito Adrian
Fong, Johnson
Jaroslavsky, Jason
Karadeniz, Derin F.
Li, Quan-Zhen
Reddy, Shantan
Roche, Aoife M.
Zhu, Chengsong
Whangbo, Jennifer S.
Dansereau, Colleen
Mackinnon, Brenda
Morris, Emily
Koo, Stephanie M.
London, Wendy B.
Baris, Safa
Ozen, Ahmet
Karakoc-Aydiner, Elif
Despotovic, Jenny M.
Satter, Lisa R. Forbes
Saitoh, Akihiko
Aizawa, Yuta
King, Alejandra
Nguyen, Mai Anh Thi
Vu, Vy Do Uyen
Snapper, Scott B.
Galy, Anne
Notarangelo, Luigi D.
Bushman, Frederic D.
Williams, David A.
Pai, Sung-Yun
BLOOD, 2023, 142 (15) : 1281 - 1296
[2] Hematopoietic Stem Cell Gene Therapy For Wiskott-Aldrich Syndrome
Witzel, Maximilian G. W.
Braun, Christian J.
Boztug, Kaan
Paruzynski, Anna
Albert, Michael H.
Schwarzer, Adrian
Modlich, Ute
Beier, Rita
Goehring, Gudrun
Naundorf, Sonja
Kuehlcke, Klaus
Rose, Martina
Fraser, Christopher
Mathias, Liesl
Ferrari, Rudolf
Abboud, Miguel R.
Al-Herz, Waleed
Kondratenko, Irina
Marodi, Laszlo
Schlegelberger, Brigitte
Baum, Christopher
Schmidt, Manfred
von Kalle, Christof
Klein, Christoph
BLOOD, 2013, 122 (21)
[3] Hematopoietic stem cell gene therapy of Wiskott-Aldrich syndrome
Boztug, Kaan
Dewey, Ricardo A.
Diez, Ines Avedillo
Schmidt, Manfred
Schwarzwaelder, Kerstin
Diestelhorst, Jana
Naundorf, Sonja
Kuehlcke, Klaus
Kondratenko, Irina
Marodi, Laszlo
von Kalle, Christoph
Welte, Karl
Klein, Christoph
HUMAN GENE THERAPY, 2007, 18 (10) : 961 - 961
[4] Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome
Boztug, Kaan
Dewey, Ricardo A.
Diez, Ines Avedillo
Manfred, Schmidt
Kerstin, Schwarzwaelder
Jana, Diestelhorst
Sonja, Naundorf
Klaus, Kuehlke
Irina, Kondratenko
Laszlo, Marodi
von Kalle, Christof
Welte, Karl
Klein, Christoph
BLOOD, 2007, 110 (11) : 155A - 155A
[5] Correction of Wiskott-Aldrich Syndrome by Hematopoietic Stem Cell Gene Therapy
Boztug, Kaan
Schmidt, Manfred
Schwarzer, Adrian
Banerjee, Pinaki
Boehm, Marie
Beier, Rita
Diez, Ines Avedillo
Dewey, Ricardo
Ball, Claudia R.
Nowrouzi, Ali
Naundorf, Sonja
Kuehlcke, Klaus
Blasczykf, Rainer
Rose, Martina
Fraser, Christopher
Liesl, Mathias
Ferrari, Rudolf
Abboud, Miguel R.
Al-Herz, Waleed
Kondratenko, Irina
Marodi, Laszlo
Orange, Jordan
Christof, von Kalle
Klein, Christoph
BLOOD, 2010, 116 (21) : 4 - 5
[6] Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome
Labrosse, Roxane
Chu, Julia
Armant, Myriam
van der Spek, Jet
Miggelbrink, Alexandra
Fong, Johnson
Everett, John K.
Raymond, Hayley
Kessler, Lyanna
Dansereau, Colleen
Mackinnon, Brenda
Koo, Stephanie
Morris, Emily
London, Wendy B.
Ozen, Ahmet
Baris, Safa
Despotovic, Jenny M.
Forbes, Lisa
Saitoh, Akihiko
Takachi, Takayuki
King, Alejandra
Thi Mai Anh Thi Nguyen
Vy Do Uyen Vu
Bushman, Frederic D.
Galy, Anne
Notarangelo, Luigi
Williams, David A.
Pai, Sung-Yun
BLOOD, 2019, 134
[7] Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome
Boztug, Kaan
Dewey, Ricardo A.
Klein, Christoph
CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2006, 8 (05) : 390 - 395
[8] Correction of Wiskott-Aldrich syndrome by hematopoietic stem cell gene therapy
Boztug, Kaan
Schmidt, Manfred
Schwarzer, Adrian
Banerjee, Pinaki P.
Boehm, Marie
Beier, Rita
Diez, Ines Avedillo
Dewey, Ricardo
Ball, Claudia R.
Nowrouzi, Ali
Naundorf, Sonja
Kuehlcke, Klaus
Blasczyk, Rainer
Rose, Martina
Fraser, Chris
Liesl, Mathias
Ferrari, Rudolf
Abboud, Miguel
Al-Herz, Waleed
Kondratenko, Irina
Marodi, Laszlo
Orange, Jordan S.
von Kalle, Christof
Klein, Christoph
HUMAN GENE THERAPY, 2010, 21 (10) : 1362 - 1363
[9] Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
Aiuti, Alessandro
Biasco, Luca
Scaramuzza, Samantha
Ferrua, Francesca
Cicalese, Maria Pia
Baricordi, Cristina
Dionisio, Francesca
Calabria, Andrea
Giannelli, Stefania
Castiello, Maria Carmina
Bosticardo, Marita
Evangelio, Costanza
Assanelli, Andrea
Casiraghi, Miriam
Di Nunzio, Sara
Callegaro, Luciano
Benati, Claudia
Rizzardi, Paolo
Pellin, Danilo
Di Serio, Clelia
Schmidt, Manfred
Von Kalle, Christof
Gardner, Jason
Mehta, Nalini
Neduva, Victor
Dow, David J.
Galy, Anne
Miniero, Roberto
Finocchi, Andrea
Metin, Ayse
Banerjee, Pinaki P.
Orange, Jordan S.
Galimberti, Stefania
Valsecchi, Maria Grazia
Biffi, Alessandra
Montini, Eugenio
Villa, Anna
Ciceri, Fabio
Roncarolo, Maria Grazia
Naldini, Luigi
SCIENCE, 2013, 341 (6148) : 865 - U71
[10] Hematopoietic Stem Cell Gene Editing for the Treatment of Wiskott-Aldrich Syndrome
Cavazza, Alessia
Rai, Rajeev
Santilli, Giorgia
Thrasher, Adrian J.
MOLECULAR THERAPY, 2018, 26 (05) : 3 - 4

← 1 2 3 4 5 →