Discovery and Early Clinical Development of the First Allele-Specific Stereopure Antisense Oligonucleotide Drug to Treat Huntington disease

被引：0

作者：

Meena ^{[1
]}

Zboray, Lori ^{[2
]}

Monteys, Alex Mas ^{[2
]}

Mohapatra, Susovan ^{[1
]}

Svrzikapa, Nenad ^{[1
]}

Lonkar, Pallavi ^{[1
]}

Morris, Aaron ^{[1
]}

Sokolovska, Anna ^{[1
]}

Yang, Hailin ^{[1
]}

Iwamoto, Naoki ^{[1
]}

Butler, David ^{[1
]}

Bowman, Keith ^{[1
]}

Standley, Stephany ^{[1
]}

Mamoru, Shimizu ^{[1
]}

Frank-Kamenetsky, Maria ^{[1
]}

Francis, Chris ^{[1
]}

Davidson, Beverley L. ^{[2
]}

Vargeese, Chandra ^{[1
]}

机构：

[1] WAVE Life Sci, Cambridge, MA USA

[2] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

来源：

NEUROTHERAPEUTICS | 2017年 / 14卷 / 01期

关键词：

D O I：

暂无

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

引用

页码：236 / 237

页数：2

共 6 条

[1] Selectivity and Biodistribution of WVE-120102, a Stereopure Allele-Specific Antisense Oligonucleotide in Development for the Treatment of Huntington's Disease
Hung, Serena
Me, Meena
Davis, John W., II
Bowman, Keith
Butler, David C. D.
Desai, Foram
Guo, Lanikai
Iwamoto, Naoki
Jang, Hyun
Frank-Kamenetsky, Maria
Mohapatra, Susovan
Shimizu, Mamoru
Standley, Stephany
Svrzikapa, Nenad
Yang, Hailin
Zhang, Jason
Zhong, Zhong
Francis, Chris
Panzara, Michael
Vargeese, Chandra
NEUROTHERAPEUTICS, 2018, 15 (01) : 247 - 247
[2] Development of allele-specific antisense oligonucleotide therapy for fibrodysplasia ossificans progressiva
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Anwar, Saeed
Quynh Nguyen
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Graf, Daniel
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JOURNAL OF BONE AND MINERAL RESEARCH, 2023, 38 : 238 - 238
[3] Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients
Skotte, Niels H.
Southwell, Amber L.
Ostergaard, Michael E.
Carroll, Jeffrey B.
Warby, Simon C.
Doty, Crystal N.
Petoukhov, Eugenia
Vaid, Kuljeet
Kordasiewicz, Holly
Watt, Andrew T.
Freier, Susan M.
Hung, Gene
Seth, Punit P.
Bennett, C. Frank
Swayze, Eric E.
Hayden, Michael R.
PLOS ONE, 2014, 9 (09):
[4] Discovery and Early Clinical Development of ISIS-HTTRx, the First HTT-Lowering Drug to Be Tested in Patients with Huntington's Disease
Leavitt, Blair
Tabrizi, Sarah
Kordasiewicz, Holly
Landwehrmeyer, Bernhard
Henry, Scott
Zanardi, Tom
Swayze, Eric
Norris, Daniel
Smith, Anne
Lane, Roger
Bennett, Frank
NEUROLOGY, 2016, 86
[5] Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene/Allele-Specific Silencing of Mutant Huntingtin
Carroll, Jeffrey B.
Warby, Simon C.
Southwell, Amber L.
Doty, Crystal N.
Greenlee, Sarah
Skotte, Niels
Hung, Gene
Bennett, C. Frank
Freier, Susan M.
Hayden, Michael R.
MOLECULAR THERAPY, 2011, 19 (12) : 2178 - 2185
[6] Development of an allele-specific minimal residual disease assay for patients with juvenile myelomonocytic leukemia-moving beyond clinical assessment.
Archambeault, Sophie
Yoshimi, Ayami
Kratz, Christian
Reising, Miriam
Fischer, Alexandra
Noellke, Peter
Locatelli, Franco
Sedlacek, Petr
Flotho, Christian
Zecca, Marco
Emanuel, Peter Dean
Castleberry, Robert
Niemeyer, Charlotte Marie
Bader, Peter
Loh, Mignon
BLOOD, 2006, 108 (11) : 758A - 758A

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