A framework for individualized splice-switching oligonucleotide therapy - Nature

New method used to develop RNA therapy for the treatment of rarediseases nature

We thank the families of our patients for their participation, trust and partnership. We also thank those who contributed advice and expertise to this project, including: B. Darras, T. Crawford, H. Lederman, A.M.R. Taylor, J. Douville, L. Black, S. Goldkind, M. Pendergast, F. Bennett, K. Tyndall, C. Reed, M. Moore, S. Agrawal, A. Krieg and colleagues in the Division of Neurology from the Center for Drug Evaluation and Research at the FDA. We thank Invitae for providing RNA-seq and B. Addy and L.

Institutional Center for Clinical and Translational Research, Boston Children’s Hospital, Boston, MA, USAJennifer Karlin Thornton & Brad Margus Department of Anesthesiology, Critical Care and Pain Medicine, Boston Children’s Hospital, Boston, MA, USACenter for Bioethics, Harvard Medical School, Boston, MA, USAPeter J. ParkEric and Wendy Schmidt Center, Broad Institute of MIT and Harvard, Cambridge, MA, USA

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