This meeting took place in 2019



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Protein Replacement through Nucleic Acid Therapies (L3)


Organizer(s) Pad Chivukula, Jean Bennett and Paloma Giangrande
April 7—10, 2019
Steamboat Grand • Steamboat Springs, Colorado USA
Discounted Abstract Deadline: Jan 8, 2019
Abstract Deadline: Feb 5, 2019
Scholarship Deadline: Jan 8, 2019
Discounted Registration Deadline: Feb 5, 2019

Sponsored by Moderna, Novo Nordisk A/S and Sarepta Therapeutics

Summary of Meeting:
Protein replacement therapy based on recombinant protein has so far been limited to genetic diseases in which the mutated protein acts extracellularly. Nucleic acid therapies such as gene therapy and messenger RNA enable replacement of intracellular proteins, or extracellular proteins too complex to manufacture. This opens up the potential to treat many previously unapproachable diseases. After early setbacks in gene therapy, a new generation of therapeutics are showing progress in the clinic. Novel messenger RNA and RNA delivery technologies are also in development, the potential of which is only beginning to be demonstrated. These represent exciting areas of therapeutic development which also touch on fundamental questions about regulation of gene expression, protein production, and immunity. This conference brings these communities together to discuss common challenges and complementarities, providing an opportunity for cross-fertilization.

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Scholarships/Awards

National Center for Advancing Translational Sciences (NCATS)/ National Eye Institute (NEI) Scholarship Recipients

Liangliang Hao
Massachusetts Institute of Technology, USA

Dong Hyun Jo
Seoul National University Hospital, South Korea

John H. Lockhart
University of South Florida, USA

Nida Mubin
Aligarh Muslim University, India

Jeffrey Rubin
Mayo Clinic Graduate School of Biomedical Sciences, USA

Arnab Rudra
Massachusetts Institute of Technology, USA