Publications

Poster – R. Blumenthal, M. Spellman. A randomized trial to determine the efficacy and safety of FCX-007 in the treatment  of recessive dystrophic epidermolysis bullosa (DEFI-RDEB). Presented at the 25^th Annual Meeting of the American Society of Gene and Cell Therapy, Washington, DC, May 17, 2022.

Poster – M. P. Marinkovich, K. J. Sridhar, S. Bagci, J. A. M. Dolorito, D. R. Keene, M. Yonchek, R Blumenthal, M Spellman, MD. The treatment of wounds associated with recessive dystrophic epidermolysis bullosa with local injections of gene-corrected, collagen VII-expressing autologous human dermal fibroblasts. Presented at the 25^th Annual Meeting of the American Society of Gene and Cell Therapy, Washington, DC, May 17, 2022.

Poster – M. P. Marinkovich, MD, A. Lane, MD, K. Sridhar, PhD, D. Keene, BS, A. Malyala, PhD, M. Spellman, MD, and J. Maslowski, MS. A Phase 1/2 Study of Genetically-Corrected,  Collagen VII Expressing Autologous Human Dermal Fibroblasts Injected into the Skin of Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB).  Presented at EB 2020 Congress, London, United Kingdom, January 2020.

Poster – M. P. Marinkovich, MD, N. Ehsani-Chimeh, MD, N. Nguyen, S. Moncrief, PhD, V. K. Dailey, M. Chakiath, A. Elayadi, PhD, S. Krishnan, MS, and J. Maslowski, MS. Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa. Presented at the American Society of Human Genetics Annual Meeting, Baltimore, Maryland, October 8, 2015.

Poster – V.K. Dailey, M. Chakiath, A. Elayadi, PhD, S. Krishnan, MS, J. Maslowski, MS, M. P. Marinkovich, MD. Development of a Genetically-Modified Human Dermal Fibroblast for the Treatment of Recessive Dystrophic Epidermolysis Bullosa. Presented at the European Society of Human Genetics, Glasgow, Scotland, United Kingdom, June 8, 2015.

Clara T Nicolas, Caitlin J VanLith, Kari L Allen, Raymond D Hickey, Zeji Du, Lori G Hillin, Rebekah M Guthman, William J Cao, Aditya Bhagwate, Daniel O’Brien, Jean-Pierre Kocher, Robert A Kaiser, Stephen J Russell, Joseph B Lillegard. In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions. bioRxiv 2021.01.02.425079; doi: https://doi.org/10.1101/2021.01.02.425079.

Thompson WS, Mondal G, Vanlith CJ, Kaiser RA, Lillegard JB. The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism. Expert Opin Orphan Drugs.2020;8(7):245-256. doi: 10.1080/21678707.2020.1791082. Epub 2020 Jul 21. PMID:33224636; PMCID: PMC7676758.

Nicolas CT, Kaiser RA, Hickey RD, Allen KL, Du Z, VanLith CJ, Guthman RM, Amiot B, Suksanpaisan L, Han B, Francipane MG, Cheikhi A, Jiang H, Bansal A, Pandey MK, Garg I, Lowe V, Bhagwate A, O’Brien D, Kocher JA, DeGrado TR, Nyberg SL, Lagasse E, Lillegard JB. Ex Vivo Cell Therapy by Ectopic Hepatocyte Transplantation Treats the Porcine Tyrosinemia Model of Acute Liver Failure. Mol Ther Methods Clin Dev. 2020 Jul 10;18:738-750. doi: 10.1016/j.omtm.2020.07.009. PMID: 32913881; PMCID: PMC7452193.

Kaiser RA, Nicolas CT, Allen KL, Chilton JA, Du Z, Hickey RD, Lillegard JB. Hepatotoxicity and Toxicology of In Vivo Lentiviral Vector Administration in Healthy and Liver-Injury Mouse Models. Hum Gene Ther Clin Dev. 2019 Jun;30(2):57-66. doi: 10.1089/humc.2018.249. Epub 2019 Apr 11. PMID: 30860398; PMCID: PMC6589498.

Hickey RD, Nicolas CT, Allen K, Mao S, Elgilani F, Glorioso J, Amiot B, VanLith C, Guthman R, Du Z, Chen H, Harding CO, Kaiser RA, Nyberg SL, Lillegard JB. Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1. Cell Transplant. 2019 Jan;28(1):79-88. doi: 10.1177/0963689718814188. Epub 2018 Nov 26. PMID: 30477316; PMCID: PMC6322137.