University of Sydney & Children's Medical Research Institutes

Assoc. Prof. Dr. Leszek Lisowski and Dr. Wendy Gold, working on gene therapy.

National Institute of Chemistry Slovenia

Prof. dr. Roman Jerala and his team, working on gene therapy.

University Medical Centre Ljubljana, Children's Hospital

Assoc. prof. Dr. Damjan Osredkar is leading the Natural History Study.

Hugh Kaul Precision Medicine Institute, The University of Alabama at Birmingham

Prof. Dr. Matt Might is leading the program for developing different models for application in the gene therapy programs.