Palmquist, Rachel
Jenkins, Sabrina Malone
Bentley, Dawn
Miller, Christine
Mao, Rong
Meibos, Bailey
Bayrak-Toydemir, Pinar
Tvrdik, Tatiana
Nadauld, Lincoln D.
Bleyl, Steven B.
Chowdhury, Shimul
Ostrander, Betsy
Flores-Daboub, Josue
Longo, Nicola
Tristani-Firouzi, Martin
Hobbs, Charlotte
Bonkowsky, Joshua L.
Brunelli, Luca
Article History
Received: 4 August 2021
Revised: 17 January 2022
Accepted: 20 January 2022
First Online: 3 February 2022
Competing interests
: J.L.B. has served as a consultant to Bluebird Bio, Inc., Calico, Inc., Denali, Neurogene, Inc., Enzyvant, Inc., and Passage Bio and owns stock in Orchard Therapeutics. S.B.B. holds stock options in Genome Medical, Inc. There are no other disclosures.
: The analysis and publication of data related to this program were approved by the Institutional Review Boards at the University of Utah and Primary Children’s Hospital with a waiver of consent and authorization. Informed consent for the clinical test was obtained in all cases from at least one parent.