Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

Autor: Yasaman Ataei, Larry Park, D. James Surmeier, B. Joseph Vu, Seung Kwak, Richard T. Surosky, Anand Narayanan, David A. Shivak, Josee Laganiere, Christer Halldin, Andrea Varrone, Matthew C. Mendel, Karsten Tillack, Lei Zhang, Bryan Zeitler, Dmitry Guschin, Lexi Kopan, Sarah J. Hinkley, Kimberly Marlen, Jocelynn R. Pearl, Qi Yu, Taneli Heikkinen, Annette Gärtner, Yalda Sedaghat, Christina Thiede, Miklós Tóth, Jennifer M. Cherone, David Paschon, Jyothisri Kondapalli, Andrea E. Kudwa, Ladislav Mrzljak, Rainier Amora, Kimmo Lehtimäki, Edward J. Rebar, Lenke Tari, Ignacio Munoz-Sanjuan, Jeffrey C. Miller, Sylvie Ramboz, Marie Svedberg, Steven Froelich, Irina Ankoudinova, Philip D. Gregory, Stephen Lam, Michelle Day, Jonathan Bard, Hoang Oanh B. Nguyen, Fyodor D. Urnov, Davis Li, Jenny Haggkvist, H. Steve Zhang, Guijuan Qiao

Publikováno v: Nature Medicine. 25:1131-1142

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 importa

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::d19307c1ae395108f0cf1b74fb590fb6
https://doi.org/10.1038/s41591-019-0478-3

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease

Autor: Bryan, Zeitler, Steven, Froelich, Kimberly, Marlen, David A, Shivak, Qi, Yu, Davis, Li, Jocelynn R, Pearl, Jeffrey C, Miller, Lei, Zhang, David E, Paschon, Sarah J, Hinkley, Irina, Ankoudinova, Stephen, Lam, Dmitry, Guschin, Lexi, Kopan, Jennifer M, Cherone, Hoang-Oanh B, Nguyen, Guijuan, Qiao, Yasaman, Ataei, Matthew C, Mendel, Rainier, Amora, Richard, Surosky, Josee, Laganiere, B Joseph, Vu, Anand, Narayanan, Yalda, Sedaghat, Karsten, Tillack, Christina, Thiede, Annette, Gärtner, Seung, Kwak, Jonathan, Bard, Ladislav, Mrzljak, Larry, Park, Taneli, Heikkinen, Kimmo K, Lehtimäki, Marie M, Svedberg, Jenny, Häggkvist, Lenke, Tari, Miklós, Tóth, Andrea, Varrone, Christer, Halldin, Andrea E, Kudwa, Sylvie, Ramboz, Michelle, Day, Jyothisri, Kondapalli, D James, Surmeier, Fyodor D, Urnov, Philip D, Gregory, Edward J, Rebar, Ignacio, Muñoz-Sanjuán, H Steve, Zhang

Publikováno v: Nature medicine. 25(7)

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

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::4d3620bad8fe21aa64bd37ef53578561
https://pubmed.ncbi.nlm.nih.gov/31263285

Improved specificity of TALE-based genome editing using an expanded RVD repertoire

Autor: Irina Ankoudinova, Philip D. Gregory, Gladys P Dulay, Fyodor D. Urnov, Stephen Lam, Sarah J. Hinkley, Jeffrey C. Miller, John J Campo, Elo Leung, Joshua E. Babiarz, Kyle A. Barlow, Danny F Xia, Rainier Amora, Anna I Vincent, Edward J. Rebar, David Paschon, Dmitry Guschin, David A. Shivak, Jinwon D Kim, Xiangdong Meng, Lei Zhang

Publikováno v: Nature methods. 12(5)

Transcription activator-like effector (TALE) proteins have gained broad appeal as a platform for targeted DNA recognition, largely owing to their simple rules for design. These rules relate the base specified by a single TALE repeat to the identity o

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d45ad9df03cdfd158cb77851257a62e4
https://pubmed.ncbi.nlm.nih.gov/25799440

Targeted genome editing across species using ZFNs and TALENs

Autor: Lei Zhang, Te-Wen Lo, Bryan Zeitler, Edward J. Ralston, Andrew H. Lee, Barbara J Meyer, Catherine S. Pickle, Andrew J. Wood, Elo Leung, Edward J. Rebar, Rainier Amora, Philip D. Gregory, Fyodor D. Urnov, Xiangdong Meng, Jeffrey C. Miller

Publikováno v: Wood, A, Lo, T-W, Zeitler, B, Pickle, C S, Ralston, E J, Lee, A H, Amora, R, Miller, J C, Leung, E, Meng, X, Zhang, L, Rebar, E J, Gregory, P D, Urnov, F D & Meyer, B J 2011, ' Targeted genome editing across species using ZFNs and TALENs ', Science, vol. 333, no. 6040, pp. 307 . https://doi.org/10.1126/science.1207773
Wood, AJ; Lo, TW; Zeitler, B; Pickle, CS; Ralston, EJ; Lee, AH; et al.(2011). Targeted genome editing across species using ZFNs and TALENs. Science, 333(6040), 307. doi: 10.1126/science.1207773. UC Berkeley: Retrieved from: http://www.escholarship.org/uc/item/4nn2f895

Evolutionary studies necessary to dissect diverse biological processes have been limited by the lack of reverse genetic approaches in most organisms with sequenced genomes. We established a broadly applicable strategy using zinc finger nucleases (ZFN

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::68707b82e2dc8f6a21bbe05e257b9c74
https://pubmed.ncbi.nlm.nih.gov/21985000