Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
About this item
Full title
Author / Creator
Zeitler, Bryan , Froelich, Steven , Marlen, Kimberly , Shivak, David A , Yu, Qi , Li, Davis , Pearl, Jocelynn R , Miller, Jeffrey C , Zhang, Lei , Paschon, David E , Hinkley, Sarah J , Ankoudinova, Irina , Lam, Stephen , Guschin, Dmitry , Kopan, Lexi , Cherone, Jennifer M , Nguyen, Hoang-Oanh B , Qiao, Guijuan , Ataei, Yasaman , Mendel, Matthew C , Amora, Rainier , Surosky, Richard , Laganiere, Josee , Vu, B Joseph , Narayanan, Anand , Sedaghat, Yalda , Tillack, Karsten , Thiede, Christina , Gärtner, Annette , Kwak, Seung , Bard, Jonathan , Mrzljak, Ladislav , Park, Larry , Heikkinen, Taneli , Lehtimäki, Kimmo K , Svedberg, Marie M , Häggkvist, Jenny , Tari, Lenke , Tóth, Miklós , Varrone, Andrea , Halldin, Christer , Kudwa, Andrea E , Ramboz, Sylvie , Day, Michelle , Kondapalli, Jyothisri , Surmeier, D James , Urnov, Fyodor D , Gregory, Philip D , Rebar, Edward J , Muñoz-Sanjuán, Ignacio and Zhang, H Steve
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
Formats
Publication information
Publisher
New York: Nature Publishing Group US
Subjects
More information
Scope and Contents
Contents
Huntington’s disease (HD) is a dominantly inherited neurodegenerative disorder caused by a CAG trinucleotide expansion in the
huntingtin
gene (
HTT
), which codes for the pathologic mutant HTT (mHTT) protein. Since normal HTT is thought to be important for brain function, we engineered zinc finger protein transcription factors (ZFP-TFs)...
Alternative Titles
Full title
Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease
Authors, Artists and Contributors
Author / Creator
Froelich, Steven
Marlen, Kimberly
Shivak, David A
Yu, Qi
Li, Davis
Pearl, Jocelynn R
Miller, Jeffrey C
Zhang, Lei
Paschon, David E
Hinkley, Sarah J
Ankoudinova, Irina
Lam, Stephen
Guschin, Dmitry
Kopan, Lexi
Cherone, Jennifer M
Nguyen, Hoang-Oanh B
Qiao, Guijuan
Ataei, Yasaman
Mendel, Matthew C
Amora, Rainier
Surosky, Richard
Laganiere, Josee
Vu, B Joseph
Narayanan, Anand
Sedaghat, Yalda
Tillack, Karsten
Thiede, Christina
Gärtner, Annette
Kwak, Seung
Bard, Jonathan
Mrzljak, Ladislav
Park, Larry
Heikkinen, Taneli
Lehtimäki, Kimmo K
Svedberg, Marie M
Häggkvist, Jenny
Tari, Lenke
Tóth, Miklós
Varrone, Andrea
Halldin, Christer
Kudwa, Andrea E
Ramboz, Sylvie
Day, Michelle
Kondapalli, Jyothisri
Surmeier, D James
Urnov, Fyodor D
Gregory, Philip D
Rebar, Edward J
Muñoz-Sanjuán, Ignacio
Zhang, H Steve
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_swepub_primary_oai_swepub_ki_se_478688
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_swepub_primary_oai_swepub_ki_se_478688
Other Identifiers
ISSN
1078-8956
E-ISSN
1546-170X
DOI
10.1038/s41591-019-0478-3
