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Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

Brief Report
Nisha Nair, Melvin Y. Rincon, Hanneke Evens, Shilpita Sarcar, Sumitava Dastidar, Emira Samara-Kuko, Omid Ghandeharian, Hiu Man Viecelli, Beat Thöny, Pieter De Bleser, Thierry VandenDriessche, Marinee K. Chuah
Journal: Blood
Blood (2014) 123 (20): 3195–3199.
https://doi.org/10.1182/blood-2013-10-534032
Published: 2014
Includes: Supplemental data