Development of an mRNA replacement therapy for phenylketonuria

by Carlos G. Perez-Garcia, Ramon Diaz-Trelles, Jerel Boyd Vega, Yanjie Bao, Marciano Sablad, Patty Limphong, Simon Chikamatsu, Hailong Yu, Wendy Taylor, Priya P. Karmali, Kiyoshi Tachikawa, Padmanabh Chivukula
Published 2022-06-01

Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model

by Ramon Diaz-Trelles, Sharon Lee, Kristen Kuakini, Jenny Park, Adrian Dukanovic, Jose A. Gonzalez, Thanhchau Dam, Jae Heon Kim, Jerel Boyd Vega, Marciano Sablad, Priya P. Karmali, Kiyoshi Tachikawa, Padmanabh Chivukula
Published 2022-09-01

Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS

by Tomoki Hirunagi, Kentaro Sahashi, Kiyoshi Tachikawa, Angel I. Leu, Michelle Nguyen, Rajesh Mukthavaram, Priya P. Karmali, Padmanabh Chivukula, Genki Tohnai, Madoka Iida, Kazunari Onodera, Manabu Ohyama, Yohei Okada, Hideyuki Okano, Masahisa Katsuno
Published 2021-06-01

Restoring ornithine transcarbamylase (OTC) activity in an OTC‐deficient mouse model using LUNAR‐OTC mRNA

by Hailong Yu, Edward Brewer, Michael Shields, Michael Crowder, Cristiano Sacchetti, Benchawanna Soontornniyomkij, Dandan Dou, Brenda Clemente, Marciano Sablad, Padmanabh Chivukula, Steve Hughes, Scott Roberts, Kumar Rajappan, Steve Tannis, Rose Sekulovich, Suezanne Parker, Pattraranee Limphong
Published 2022-06-01

Anti-HFRS Human IgG Produced in Transchromosomic Bovines Has Potent Hantavirus Neutralizing Activity and Is Protective in Animal Models

by Casey C. Perley, Rebecca L. Brocato, Hua Wu, Christoph Bausch, Priya P. Karmali, Jerel B. Vega, Melanie V. Cohen, Brandon Somerville, Steven A. Kwilas, Lucia M. Principe, Joshua Shamblin, Padmanabh Chivukula, Eddie Sullivan, Jay W. Hooper
Published 2020-05-01

Hepatocyte-specific activity of TSC22D4 triggers progressive NAFLD by impairing mitochondrial function

by Gretchen Wolff, Minako Sakurai, Amit Mhamane, Maria Troullinaki, Adriano Maida, Ioannis K. Deligiannis, Kelvin Yin, Peter Weber, Jakob Morgenstern, Annika Wieder, Yun Kwon, Revathi Sekar, Anja Zeigerer, Michael Roden, Matthias Blüher, Nadine Volk, Tanja Poth, Thilo Hackert, Lena Wiedmann, Francesca De Angelis Rigotti, Juan Rodriguez-Vita, Andreas Fischer, Rajesh Mukthavaram, Pattraranee Limphong, Kiyoshi Tachikawa, Priya Karmali, Joseph Payne, Padmanabh Chivukula, Bilgen Ekim-Üstünel, Celia P. Martinez-Jimenez, Julia Szendrödi, Peter Nawroth, Stephan Herzig
Published 2022-06-01