PublicationsA framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseasesOlivia Kim-McManus, Joseph G. Gleeson, Laurence Mignon, Amena Smith Fine, Winston Yan, Nicole Nolen, Scott Demarest, Elizabeth...
Illustrated ExamplesPositive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies
Articles & PodcastsN=1 Collaborative Welcomes Hugh Hempel as Executive Director to Lead the Next Phase of Growth
