Mannucci, Ilaria
Dang, Nghi D. P.
Huber, Hannes
Murry, Jaclyn B.
Abramson, Jeff
Althoff, Thorsten
Banka, Siddharth
Baynam, Gareth
Bearden, David
Beleza-Meireles, Ana
Benke, Paul J.
Berland, Siren
Bierhals, Tatjana
Bilan, Frederic
Bindoff, Laurence A.
Braathen, Geir Julius
Busk, Øyvind L.
Chenbhanich, Jirat
Denecke, Jonas
Escobar, Luis F.
Estes, Caroline
Fleischer, Julie
Groepper, Daniel
Haaxma, Charlotte A.
Hempel, Maja
Holler-Managan, Yolanda
Houge, Gunnar
Jackson, Adam
Kellogg, Laura
Keren, Boris
Kiraly-Borri, Catherine
Kraus, Cornelia
Kubisch, Christian
Le Guyader, Gwenael
Ljungblad, Ulf W.
Brenman, Leslie Manace
Martinez-Agosto, Julian A.
Might, Matthew
Miller, David T.
Minks, Kelly Q.
Moghaddam, Billur
Nava, Caroline
Nelson, Stanley F.
Parant, John M.
Prescott, Trine
Rajabi, Farrah
Randrianaivo, Hanitra
Reiter, Simone F.
Schuurs-Hoeijmakers, Janneke
Shieh, Perry B.
Slavotinek, Anne
Smithson, Sarah
Stegmann, Alexander P. A.
Tomczak, Kinga
Tveten, Kristian
Wang, Jun
Whitlock, Jordan H.
Zweier, Christiane
McWalter, Kirsty
Juusola, Jane
Quintero-Rivera, Fabiola
Fischer, Utz
Yeo, Nan Cher
Kreienkamp, Hans-Jürgen
Lessel, Davor https://orcid.org/0000-0003-4496-244X
Funding for this research was provided by:
Deutsche Forschungsgemeinschaft (LE4223/1-1, Kr1321/8-2)
National Institutes of Health (U54 OD030167)
Universitätsklinikum Hamburg-Eppendorf (UKE)
Article History
Received: 2 September 2020
Accepted: 28 April 2021
First Online: 21 May 2021
Declarations
:
: The study was performed in accordance with the Declaration of Helsinki protocols. Written informed consent for all 25 subjects was obtained from the parents or legal guardians in accordance with protocols approved by the Ethics Committee of the Hamburg Chamber of Physicians: PV 3802 and the University of California, Los Angeles (UCLA) IRB: 11-001087. Zebrafish were maintained according to protocols by the University of Alabama Zebrafish Research Facility (ZRF) Animal Resources Program which maintains full Association for Assessment and Accreditation of Laboratory Animal Care International (AAALAC) accreditation and is assured with the Office of Laboratory Animal Welfare (OLAW). All zebrafish studies followed protocols approved by the University of Alabama Institutional Animal Care and Use Committee (IACUC): APN22158.
: A written consent was obtained from the parents or legal guardians to publish the details of all 25 affected individuals.
: K.M. and J.J. are employees of GeneDx, Inc. The remaining authors declare that they have no competing interests.