Treatment of transthyretin (TTR) amyloid cardiomyopathy with an antisense oligonucleotide inhibitor of TTR synthesis

por Benson, Merrill D, Ackermann, Elizabeth J, Monia, Brett P
Publicado 2015

The ISIS-TTRRx-CS2 phase 3 study in patients with familial amyloid polyneuropathy: Baseline results of the first 100 patients for the NIS, NIS+7 and mNIS+7 using different methods...

por Bergemann, Rito, Ackermann, Elizabeth, Monia, Brett, Shenker, Andrew, Dyck, Peter
Publicado 2015

Genomic Analysis of wig-1 Pathways

por Sedaghat, Yalda, Mazur, Curt, Sabripour, Mahyar, Hung, Gene, Monia, Brett P.
Publicado 2012

A phase 3 clinical trial with ISIS-TTRRx, a 2nd-generation antisense oligonucleotide targeting transthyretin (TTR), for the treatment of TTR amyloid cardiomyopathy

por Millns, Helen, Bergemann, Rito, Ackermann, Elizabeth, Monia, Brett, Lukas, Mary Ann
Publicado 2015

Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR

por Sasaki, Shruti, Sun, Rachel, Bui, Huynh-Hoa, Crosby, Jeff R., Monia, Brett P., Guo, Shuling
Publicado 2019

Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations

por Huang, Lulu, Aghajan, Mariam, Quesenberry, Tianna, Low, Audrey, Murray, Susan F., Monia, Brett P., Guo, Shuling
Publicado 2019

Antisense oligonucleotide‐mediated knockdown of Mpzl3 attenuates the negative metabolic effects of diet‐induced obesity in mice

por Worley, Beth L., Auen, Thomas, Arnold, Amy C., Monia, Brett P., Hempel, Nadine, Czyzyk, Traci A.
Publicado 2021

CFTR mRNAs with nonsense codons are degraded by the SMG6-mediated endonucleolytic decay pathway

por Sanderlin, Edward J., Keenan, Melissa M., Mense, Martin, Revenko, Alexey S., Monia, Brett P., Guo, Shuling, Huang, Lulu
Publicado 2022

Antisense suppression of the nonsense mediated decay factor Upf3b as a potential treatment for diseases caused by nonsense mutations

por Huang, Lulu, Low, Audrey, Damle, Sagar S., Keenan, Melissa M., Kuntz, Steven, Murray, Susan F., Monia, Brett P., Guo, Shuling
Publicado 2018

Antisense transcripts are targets for activating small RNAs

por Schwartz, Jacob C, Younger, Scott T, Nguyen, Ngoc-Bich, Hardy, Daniel B, Monia, Brett P, Corey, David R, Janowski, Bethany A
Publicado 2008

Peripheral Reduction of FGFR4 with Antisense Oligonucleotides Increases Metabolic Rate and Lowers Adiposity in Diet-Induced Obese Mice

por Yu, Xing Xian, Watts, Lynnetta M., Manchem, Vara Prasad, Chakravarty, Kaushik, Monia, Brett P., McCaleb, Michael L., Bhanot, Sanjay
Publicado 2013

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice

por Tasfaout, Hichem, Buono, Suzie, Guo, Shuling, Kretz, Christine, Messaddeq, Nadia, Booten, Sheri, Greenlee, Sarah, Monia, Brett P., Cowling, Belinda S., Laporte, Jocelyn
Publicado 2017

Population Pharmacokinetic–Pharmacodynamic Modeling of Inotersen, an Antisense Oligonucleotide for Treatment of Patients with Hereditary Transthyretin Amyloidosis

por Yu, Rosie Z., Collins, Jon W., Hall, Shannon, Ackermann, Elizabeth J., Geary, Richard S., Monia, Brett P., Henry, Scott P., Wang, Yanfeng
Publicado 2020

Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals

por Swayze, Eric E., Siwkowski, Andrew M., Wancewicz, Edward V., Migawa, Michael T., Wyrzykiewicz, Tadeusz K., Hung, Gene, Monia, Brett P., Bennett, and C. Frank
Publicado 2007

Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease

por Guo, Shuling, Booten, Sheri L, Watt, Andrew, Alvarado, Luis, Freier, Susan M, Teckman, Jeffery H, McCaleb, Michael L, Monia, Brett P
Publicado 2014

TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis

por Ganz, Tomas, Nemeth, Elizabeta, Rivella, Stefano, Goldberg, Paul, Dibble, Andrew R., McCaleb, Michael L., Guo, Shuling, Monia, Brett P., Barrett, Terrance D.
Publicado 2023

Reduction in ocular complement factor B protein in mice and monkeys by systemic administration of factor B antisense oligonucleotide

por Grossman, Tamar R., Carrer, Michele, Shen, Lijiang, Johnson, Robert B., Hettrick, Lisa A., Henry, Scott P., Monia, Brett P., McCaleb, Michael L.
Publicado 2017

In Vitro and In Vivo Effects of SerpinA1 on the Modulation of Transthyretin Proteolysis

por Bezerra, Filipa, Niemietz, Christoph, Schmidt, Hartmut H. J., Zibert, Andree, Guo, Shuling, Monia, Brett P., Gonçalves, Paula, Saraiva, Maria João, Almeida, Maria Rosário
Publicado 2021

Transthyretin Antisense Oligonucleotides Lower Circulating RBP4 Levels and Improve Insulin Sensitivity in Obese Mice

por Zemany, Laura, Bhanot, Sanjay, Peroni, Odile D., Murray, Susan F., Moraes-Vieira, Pedro M., Castoldi, Angela, Manchem, Prasad, Guo, Shuling, Monia, Brett P., Kahn, Barbara B.
Publicado 2015

Partial Hepatectomy Induced Long Noncoding RNA Inhibits Hepatocyte Proliferation during Liver Regeneration

por Huang, Lulu, Damle, Sagar S., Booten, Sheri, Singh, Priyam, Sabripour, Mahyar, Hsu, Jeff, Jo, Minji, Katz, Melanie, Watt, Andy, Hart, Christopher E., Freier, Susan M., Monia, Brett P., Guo, Shuling
Publicado 2015