In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

1 In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions Clara T Nicolas 1,2,3 , Caitlin J VanLith 1 , Kari L Allen 1 , Raymond D Hickey 1,4 , Zeji Du 1 , Lori G Hillin 1 , Rebekah M Guthman 1,5 , William J Cao 1 , Aditya Bhagwate 6 , Daniel O’Brien 6 , Jean- Pierre Kocher 6 , Robert A Kaiser 1,7 , Stephen J Russell 4 , Joseph B Lillegard 1,7,8 1. Department of Surgery, Mayo Clinic, Rochester, MN 2. Faculty of Medicine, University of Barcelona, Barcelona, Spain 3. Department of Surgery, University of Alabama Birmingham, Birmingham, AL 4. Department of Molecular Medicine, Mayo Clinic, Rochester, MN 5. Medical College of Wisconsin, Wausau, WI 6. Department of Biomedical Statistics and Informatics, Mayo Clinic, Rochester, MN 7. Midwest Fetal Care Center, Children’s Hospitals and Clinics of Minnesota, Minneapolis, MN 8. Pediatric Surgical Associates, Minneapolis, MN Conflict of Interest Statement: The authors have declared that no conflict of interest exists. (which was not certified by peer review) is the author/funder. All rights reserved. No reuse allowed without permission. The copyright holder for this preprint this version posted March 16, 2021. ; https://doi.org/10.1101/2021.01.02.425079 doi: bioRxiv preprint

In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions

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