Zobrazeno 1 - 10
of 41
pro vyhledávání: '"Andrew T Watt"'
Autor:
Niels H Skotte, Amber L Southwell, Michael E Østergaard, Jeffrey B Carroll, Simon C Warby, Crystal N Doty, Eugenia Petoukhov, Kuljeet Vaid, Holly Kordasiewicz, Andrew T Watt, Susan M Freier, Gene Hung, Punit P Seth, C Frank Bennett, Eric E Swayze, Michael R Hayden
Publikováno v:
PLoS ONE, Vol 9, Iss 9, p e107434 (2014)
Huntington disease (HD) is an inherited, fatal neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin gene. The mutant protein causes neuronal dysfunction and degeneration resulting in motor dysfunction, cognitive decline, and
Externí odkaz:
https://doaj.org/article/df087f84149f4dfba0bfc4bdc1b0f552
Autor:
Ya-Wen Yang, Bibek Poudel, Julia Frederick, Poonam Dhillon, Rojesh Shrestha, Ziyuan Ma, Junnan Wu, Koji Okamoto, Jeffrey B. Kopp, Sheri L. Booten, Danielle Gattis, Andrew T. Watt, Matthew Palmer, Mariam Aghajan, Katalin Susztak
Publikováno v:
Mol Ther
Coding variants (named G1 and G2) in Apolipoprotein L1 (APOL1) can explain most excess risk of kidney disease observed in African American individuals. It has been proposed that risk variant APOL1 dose, such as increased risk variant APOL1 level serv
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d40676cfce2d670ef16e0e8c841d3ce9
https://europepmc.org/articles/PMC9263291/
https://europepmc.org/articles/PMC9263291/
Publikováno v:
Nucleic Acid Therapeutics
Reduction of matched and nearly complementary unintended transcripts was evaluated for 96 antisense oligonucleotides (ASOs) and 832 nearly matched unintended transcripts. The ASOs were 16–20 nucleotide “gapmers” with a gap of 8–10 DNA residue
Autor:
Paola Delbini, Maxwell Chappell, Valentina Ghiaccio, Danuta Jarocha, Coralea Stephanou, Maria Domenica Cappellini, Marina Kleanthous, Soteroula Christou, Shuling Guo, Kim Smith-Whitley, Irene Motta, Carla Casu, Alisa Dong, Deepa Manwani, Connie Chen, Susan M. Freier, Stefano Rivella, Andrew T. Watt, Laura Breda, Yasuhiro Ikawa, Raechel Peralta, Osheiza Abdulmalik, Sagar S. Damle
Publikováno v:
Haematologica
β-thalassemia is a disorder caused by altered hemoglobin protein synthesis and affects individuals worldwide. Severe forms of the disease, left untreated, can result in death before the age of 3 years (1). The standard of care consists of chronic an
Autor:
Susan M. Freier, Mats Ljungman, Lanbo Xiao, Sahr Yazdani, Kristin M. Juckette, Andrew T. Watt, Mona Batish, Shuling Guo, Alexander R. Gawronski, Saravana M. Dhanasekaran, Sudhanshu Shukla, John T. Wei, Michael Uhl, Rohit Malik, Hui Jiang, Yasuyuki Hosono, Yashar S. Niknafs, Yuanyuan Qiao, Utsav Patel, Sethuramasundaram Pitchiaya, Rohit Mehra, Lakshmi P. Kunju, Michelle T. Paulsen, Felix Y. Feng, Rolf Backofen, Xia Jiang, Xuhong Cao, Shruthi Subramaniam, Cenk Sahinalp, Tzu-Ying Liu, Jean C.-Y. Tien, Matthew K. Iyer, Girish C. Shukla, Arul M. Chinnaiyan, Yajia Zhang, Marcin Cieślik, Lisha Wang
Publikováno v:
Nature genetics, vol 50, iss 6
Nature genetics
Nature genetics
The androgen receptor (AR) plays a critical role in the development of the normal prostate as well as prostate cancer. Using an integrative transcriptomic analysis of prostate cancer cell lines and tissues, we identified ARLNC1 (AR-regulated long non
Autor:
Peter Adamson, Gene Hung, Brett P. Monia, Lisa Hettrick, Robert B. Johnson, Tamar R. Grossman, Raechel Peralta, Scott P. Henry, Michael L. McCaleb, Andrew T. Watt
Publikováno v:
Immunobiology. 221:701-708
Systemic lupus erythematosus is an autoimmune disease that manifests in widespread complement activation and deposition of complement fragments in the kidney. The complement pathway is believed to play a significant role in the pathogenesis and in th
Autor:
Andrew T. Watt, Lisa Hettrick, Cheng Cheng, Virginia Kimonis, Lac Ta, Michele Carrer, Tamar R. Grossman, Lan Weiss, Nina Raben, Mindy Chang
Publikováno v:
Molecular Genetics and Metabolism. 129:S90
Autor:
Soteroula Christou, Alisa Dong, Shuling Guo, Deepa Manwani, Laura Breda, Connie Chen, Yasuhiro Ikawa, Stefano Rivella, Susan M. Freier, Kim Smith-Whitley, Marina Kleanthous, Valentina Ghiaccio, Andrew T. Watt, Carla Casu, Irene Motta, Osheiza Abdulmalik, Raechel Peralta, Maxwell Chappell, Coralea Stephanou, Maria Domenica Cappellini, Paola Delbini
Publikováno v:
Blood. 134:2244-2244
The β thalassemia trait is associated with over 300 mutations in the β-globin gene that lead to reduced (β+ allele) or absent (β0 allele) synthesis of the β globin chain. A subset of these mutations affect the canonic splicing of the β globin m
Autor:
Holly Kordasiewicz, Michael R. Hayden, Clarence Frank Bennett, Crystal N. Doty, Erika B. Villanueva, Punit P. Seth, Kuljeet Vaid, Susan M. Freier, Niels H. Skotte, Eugenia Petoukhov, Amber L. Southwell, Jeffrey B. Carroll, Michael E. Østergaard, Eric E. Swayze, Andrew T. Watt, Yuanyun Xie
Publikováno v:
Molecular Therapy. 22:2093-2106
Huntington disease (HD) is a dominant, genetic neurodegenerative disease characterized by progressive loss of voluntary motor control, psychiatric disturbance, and cognitive decline, for which there is currently no disease-modifying therapy. HD is ca
Autor:
Xuhong Cao, Shuling Guo, Saravana M. Dhanasekaran, Sudhanshu Shukla, Yuping Zhang, Jean Tien, Sethuramasundaram Pitchaiya, Sathiya Pandi Narayanan, Susan M. Freier, Sunita Shankar, Srihari Srinivasan, Palak Shah, Andrew T. Watt, Arul M. Chinnaiyan, Felix Y. Feng, Xiaoming Wang, David G. Beer, Lanbo Xiao
Publikováno v:
Cancer Research. 79:3573-3573
Non-small cell lung cancer (NSCLC) is a leading cause of cancer-related deaths in the world. Disease subtypes include lung adenocarcinoma (LUAD), which accounts for nearly 50% of lung malignancies, and lung squamous carcinoma (LUSC). Disease diagnosi