Keller, Nancy
Midgley, Julian
Khalid, Ehtesham
Lesmana, Harry
Mathew, Georgie
Mincham, Christine
Teig, Norbert
Khan, Zubair
Khosla, Indu
Mehr, Sam
Guran, Tulay
Buder, Kathrin
Xu, Hong
Alhasan, Khalid
Buyukyilmaz, Gonul
Weaver, Nicole
Saba, Julie D. http://orcid.org/0000-0002-4894-919X
Funding for this research was provided by:
National Institute of Child Health and Human Development (1R01HD113778)
Article History
Received: 28 November 2023
Accepted: 6 August 2024
First Online: 27 September 2024
Declarations
:
: The study was conducted in accordance with the Declaration of Helsinki and approved by the Institutional Review Board of University of California San Francisco (protocol 20–32291, date of approval 03/24/2021). Informed consent was obtained from all subjects involved in the study through direct interaction with the Principal Investigator (Julie Saba). For subjects reported in the literature, the information is publicly available, and no consent was obtained.
: All authors have consented to publication of this manuscript in the current form.
: J.D.S. is an author on patent International Application Serial No. PCT/US2021/018613, “Adeno-Associated Viral (Aav)-Mediated <i>SGPL1</i> Gene Therapy for Treatment of Sphingosine-1-Phosphate Lyase Insufficiency Syndrome (SPLIS)” published on 08/26/2021 and assigned publication number WO 2021/168140. All other authors declare no conflicts of interest.