Zobrazeno 1 - 10
of 79
pro vyhledávání: '"Chandra, Vargeese"'
Autor:
Yuanjing Liu, Amy Andreucci, Naoki Iwamoto, Yuan Yin, Hailin Yang, Fangjun Liu, Alexey Bulychev, Xiao Shelley Hu, Xuena Lin, Sarah Lamore, Saurabh Patil, Susovan Mohapatra, Erin Purcell-Estabrook, Kristin Taborn, Elena Dale, Chandra Vargeese
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 28, Iss , Pp 558-570 (2022)
A large hexanucleotide (G4C2) repeat expansion in the first intronic region of C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Several mechanisms have been proposed to explain how the
Externí odkaz:
https://doaj.org/article/abb85617456d47dc9e93f622cb10832e
Autor:
Yuanjing Liu, Jean-Cosme Dodart, Helene Tran, Shaunna Berkovitch, Maurine Braun, Michael Byrne, Ann F. Durbin, Xiao Shelley Hu, Naoki Iwamoto, Hyun Gyung Jang, Pachamuthu Kandasamy, Fangjun Liu, Kenneth Longo, Jörg Ruschel, Juili Shelke, Hailin Yang, Yuan Yin, Amy Donner, Zhong Zhong, Chandra Vargeese, Robert H. Brown
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-15 (2021)
C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-assoc
Externí odkaz:
https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de
Autor:
Nenad Svrzikapa, Kenneth A. Longo, Nripesh Prasad, Ramakrishna Boyanapalli, Jeffrey M. Brown, Daniel Dorset, Scott Yourstone, Jason Powers, Shawn E. Levy, Aaron J. Morris, Chandra Vargeese, Jaya Goyal
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 162-173 (2020)
Novel treatments for Huntington’s disease (HD), a progressive neurodegenerative disorder, include selective targeting of the mutant allele of the huntingtin gene (mHTT) carrying the abnormally expanded disease-causing cytosine-adenine-guanine (CAG)
Externí odkaz:
https://doaj.org/article/65d1f0e1bbb64c3b9d640e8783a84fb1
Autor:
Wei Liu, Naoki Iwamoto, Subramanian Marappan, Khoa Luu, Snehlata Tripathi, Erin Purcell-Estabrook, Juili Dilip Shelke, Himali Shah, Anthony Lamattina, Qianli Pan, Brett Schrand, Frank Favaloro, Mugdha Bedekar, Arindom Chatterjee, Jigar Desai, Tomomi Kawamoto, Genliang Lu, Jake Metterville, Milinda Samaraweera, Priyanka Shiva Prakasha, Hailin Yang, Yuan Yin, Hui Yu, Paloma H Giangrande, Michael Byrne, Pachamuthu Kandasamy, Chandra Vargeese
Publikováno v:
Nucleic Acids Research. 51:4126-4147
Herein, we report the systematic investigation of stereopure phosphorothioate (PS) and phosphoryl guanidine (PN) linkages on siRNA-mediated silencing. The incorporation of appropriately positioned and configured stereopure PS and PN linkages to N-ace
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::f52fce2dc6c2c20d3fb621264918bd6b
https://doi.org/10.1093/nar/gkad268
https://doi.org/10.1093/nar/gkad268
Autor:
Prashant Monian, Chikdu Shivalila, Genliang Lu, Mamoru Shimizu, David Boulay, Karley Bussow, Michael Byrne, Adam Bezigian, Arindom Chatterjee, David Chew, Jigar Desai, Frank Favaloro, Jack Godfrey, Andrew Hoss, Naoki Iwamoto, Tomomi Kawamoto, Jayakanthan Kumarasamy, Anthony Lamattina, Amber Lindsey, Fangjun Liu, Richard Looby, Subramanian Marappan, Jake Metterville, Ronelle Murphy, Jeff Rossi, Tom Pu, Bijay Bhattarai, Stephany Standley, Snehlata Tripathi, Hailin Yang, Yuan Yin, Hui Yu, Cong Zhou, Luciano H. Apponi, Pachamuthu Kandasamy, Chandra Vargeese
Publikováno v:
Nature Biotechnology. 40:1093-1102
Technologies that recruit and direct the activity of endogenous RNA-editing enzymes to specific cellular RNAs have therapeutic potential, but translating them from cell culture into animal models has been challenging. Here we describe short, chemical
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0e36a5ee2c78d18679d808cc838638dd
https://doi.org/10.1038/s41587-022-01225-1
https://doi.org/10.1038/s41587-022-01225-1
Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS
Autor:
Pachamuthu Kandasamy, Yuanjing Liu, Vincent Aduda, Sandheep Akare, Rowshon Alam, Amy Andreucci, David Boulay, Keith Bowman, Michael Byrne, Megan Cannon, Onanong Chivatakarn, Juili Dilip Shelke, Naoki Iwamoto, Tomomi Kawamoto, Jayakanthan Kumarasamy, Sarah Lamore, Muriel Lemaitre, Xuena Lin, Kenneth Longo, Richard Looby, Subramanian Marappan, Jake Metterville, Susovan Mohapatra, Bridget Newman, Ik-Hyeon Paik, Saurabh Patil, Erin Purcell-Estabrook, Mamoru Shimizu, Pochi Shum, Stephany Standley, Kris Taborn, Snehlata Tripathi, Hailin Yang, Yuan Yin, Xiansi Zhao, Elena Dale, Chandra Vargeese
Publikováno v:
Nucleic Acids Research. 50:5401-5423
Attaining sufficient tissue exposure at the site of action to achieve the desired pharmacodynamic effect on a target is an important determinant for any drug discovery program, and this can be particularly challenging for oligonucleotides in deep tis
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f5bd2c8b0ca0bc373823740210c986ec
https://doi.org/10.1093/nar/gkac037
https://doi.org/10.1093/nar/gkac037
Autor:
ElenaAbbie DaleMaguire, Lankai Guo, Michael Byrne, Megan Cannon, Kris Taborn, Priyanka Shiva Prakasha, Kenneth Longo, Naoki Iwamoto, Pachamuthu Kandasamy, Mamoru Shimizu, Pochi Shum, Ashwini Ranade, Raghuvaran Iyer, Keith Bowman, Hailin Yang, Yuan Benny Yin, Fangjun Liu, Susovan Mohapatra, Lilia Macovei, Saurabh Patil, Janice Lansita, Xiao Shelley Hu, Karen Smith, Jaya Goyal, Padma Narayanan, Ken Rhodes, Michael Panzara, Chandra Vargeese
Publikováno v:
Alzheimer's & Dementia. 18
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::56470a680ebd876284149c9abf9945ca
https://doi.org/10.1002/alz.063730
https://doi.org/10.1002/alz.063730
Autor:
Pachamuthu Kandasamy, Ann Fiegen Durbin, Michael Byrne, Robert H. Brown, Jean-Cosme Dodart, Yuan Yin, Kenneth Longo, Amy Donner, Fangjun Liu, Xiao Shelley Hu, Naoki Iwamoto, Juili Dilip Shelke, Maurine Braun, Chandra Vargeese, Jörg Ruschel, Hailin Yang, Helene Tran, Shaunna Berkovitch, Yuanjing Liu, Zhong Zhong, Hyun Gyung Jang
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-15 (2021)
Nature Communications
Nature Communications
A large G4C2-repeat expansion in C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Neuronal degeneration associated with this expansion arises from a loss of C9orf72 protein, the accumu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cd5a4da0d93fe4e3773026ba3df968a1
https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de
https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de
Autor:
Daniel Dorset, Ramakrishna Boyanapalli, Kenneth Longo, Jeffrey M. Brown, Aaron J. Morris, Nripesh Prasad, Scott Yourstone, Shawn Levy, Chandra Vargeese, Nenad Svrzikapa, Jaya Goyal, Jason Powers
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 162-173 (2020)
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 162-173 (2020)
Novel treatments for Huntington’s disease (HD), a progressive neurodegenerative disorder, include selective targeting of the mutant allele of the huntingtin gene (mHTT) carrying the abnormally expanded disease-causing cytosine-adenine-guanine (CAG)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b6a876e8e2835d79e2bba28b9a1adca6
http://europepmc.org/articles/PMC7648085
http://europepmc.org/articles/PMC7648085
Autor:
Kirsten Deprey, Nefeli Batistatou, Marjoke F. Debets, Jack Godfrey, Kirstin B. VanderWall, Rebecca R. Miles, Livia Shehaj, Jiaxing Guo, Amy Andreucci, Pachamuthu Kandasamy, Genliang Lu, Mamoru Shimizu, Chandra Vargeese, Joshua A. Kritzer
Publikováno v:
ACS Chem Biol
A major obstacle to the development of effective oligonucleotide therapeutics is a lack of understanding about their cytosolic and nuclear penetration. To address this problem, we have applied the chloroalkane penetration assay (CAPA) to oligonucleot
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5ca7a1a52699e0b7e5fa1b2efc41faf6
https://europepmc.org/articles/PMC9252293/
https://europepmc.org/articles/PMC9252293/
