Funding for this research was provided by:
National Institute of Neurological Disorders and Stroke (5K08NS102398-03)
National Organization for Rare Disorders Thrasher Research Fund
Received: 24 June 2019
Accepted: 9 August 2019
First Online: 6 September 2019
: M.H.P. is a consultant and has equity interest in CRISPR Tx and Allogene Tx, but neither company had input or opinions on the subject matter described in this manuscript. The remaining authors declare no competing interests.