Topal, Joseph
Panchal, Neelam
Barroeta, Amairelys
Roppelt, Anna
Mudde, Annelotte
Gaspar, H. Bobby
Thrasher, Adrian J.
Houghton, Benjamin C.
Booth, Claire http://orcid.org/0000-0002-2626-5037
Funding for this research was provided by:
Action Medical Research (GN2761)
Medical Research Foundation
Great Ormond Street Hospital Charity
Article History
Received: 22 August 2022
Accepted: 19 October 2022
First Online: 3 November 2022
Declarations
:
: Ethical approval in place.
: Informed consent was obtained from the patient and/or parents of each patient for research samples, and patients were treated in accordance with the Declaration of Helsinki.
: HBG is an employee of Orchard Therapeutics and holds equity in the company.