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Small Molecule Splicing Modifiers with Systemic HTT-lowering Activity

Anuradha Bhattacharyya,Christopher R. Trotta,Jana Narasimhan, Kari J. Wiedinger,Wencheng Li,Kerstin A. Effenberger,Matthew G. Woll, Minakshi B. Jani,Nicole Risher,Shirley Yeh,Yaofeng Cheng,Nadiya Sydorenko,Young-Choon Moon,Gary M. Karp,Marla Weetall,Amal Dakka,Vijayalakshmi Gabbeta,Nikolai A. Naryshkin,Jason D. Graci,Thomas Tripodi Jr.,Amber Southwell,Michael Hayden,Joseph M. Colacino,Stuart W. Peltz

Nature Communications（2021）

Cited 45|Views42
Key words
Biotechnology,Cognitive neuroscience,RNA splicing,Target validation,Science,Humanities and Social Sciences,multidisciplinary
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