Gomez-Ospina, Natalia
Scharenberg, Samantha G.
Mostrel, Nathalie
Bak, Rasmus O. http://orcid.org/0000-0002-7383-0297
Mantri, Sruthi
Quadros, Rolen M.
Gurumurthy, Channabasavaiah B. http://orcid.org/0000-0002-8022-4033
Lee, Ciaran http://orcid.org/0000-0002-0789-9149
Bao, Gang
Suarez, Carlos J.
Khan, Shaukat
Sawamoto, Kazuki
Tomatsu, Shunji
Raj, Nitin
Attardi, Laura D.
Aurelian, Laure
Porteus, Matthew H. http://orcid.org/0000-0002-3850-4648
Funding for this research was provided by:
U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke (5K08NS102398-03, 5K08NS102398-03)
National Organization for Rare Disorders Thrasher Research Fund
Article History
Received: 24 June 2019
Accepted: 9 August 2019
First Online: 6 September 2019
Competing interests
: 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.