About the Authors

Lingli Li

Affiliations Cell and Gene Therapy, Murdoch Childrens Research Institute, Parkville, Victoria, Australia, Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Parkville, Victoria, Australia

Lucille Voullaire

Affiliation Cell and Gene Therapy, Murdoch Childrens Research Institute, Parkville, Victoria, Australia

Chiranjeevi Sandi

Affiliation Division of Biosciences, School of Health Sciences and Social Care, Brunel University, Uxbridge, United Kingdom

Mark A. Pook

Affiliation Division of Biosciences, School of Health Sciences and Social Care, Brunel University, Uxbridge, United Kingdom

Panos A. Ioannou

Contributed equally to this work with: Panos A. Ioannou, Martin B. Delatycki, Joseph P. Sarsero

† Deceased

Affiliations Cell and Gene Therapy, Murdoch Childrens Research Institute, Parkville, Victoria, Australia, Department of Paediatrics, The University of Melbourne, Royal Children’s Hospital, Parkville, Victoria, Australia

Martin B. Delatycki

Contributed equally to this work with: Panos A. Ioannou, Martin B. Delatycki, Joseph P. Sarsero

Affiliations Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Parkville, Victoria, Australia, Department of Paediatrics, The University of Melbourne, Royal Children’s Hospital, Parkville, Victoria, Australia, Department of Clinical Genetics, Austin Health, Heidelberg, Victoria, Australia

Joseph P. Sarsero

Contributed equally to this work with: Panos A. Ioannou, Martin B. Delatycki, Joseph P. Sarsero

joe@sarsero.com

Affiliations Cell and Gene Therapy, Murdoch Childrens Research Institute, Parkville, Victoria, Australia, Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Parkville, Victoria, Australia, Department of Paediatrics, The University of Melbourne, Royal Children’s Hospital, Parkville, Victoria, Australia

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

Conceived and designed the experiments: LL MP PAI MBD JS. Performed the experiments: LL LV CS JS. Analyzed the data: LL LV CS MP PAI MBD JS. Contributed reagents/materials/analysis tools: MP JS. Wrote the paper: JS.