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2014 4
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Phase 1/2a clinical trial of gene-corrected autologous cell therapy for recessive dystrophic epidermolysis bullosa.
Eichstadt S, Barriga M, Ponakala A, Teng C, Nguyen NT, Siprashvili Z, Nazaroff J, Gorell ES, Chiou AS, Taylor L, Khuu P, Keene DR, Rieger K, Khosla RK, Furukawa LK, Lorenz HP, Marinkovich MP, Tang JY. Eichstadt S, et al. JCI Insight. 2019 Oct 3;4(19):e130554. doi: 10.1172/jci.insight.130554. JCI Insight. 2019. PMID: 31578311 Free PMC article. Clinical Trial.
Gene therapy for skin diseases.
Gorell E, Nguyen N, Lane A, Siprashvili Z. Gorell E, et al. Cold Spring Harb Perspect Med. 2014 Apr 1;4(4):a015149. doi: 10.1101/cshperspect.a015149. Cold Spring Harb Perspect Med. 2014. PMID: 24692191 Free PMC article. Review.
Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa.
Sebastiano V, Zhen HH, Haddad B, Bashkirova E, Melo SP, Wang P, Leung TL, Siprashvili Z, Tichy A, Li J, Ameen M, Hawkins J, Lee S, Li L, Schwertschkow A, Bauer G, Lisowski L, Kay MA, Kim SK, Lane AT, Wernig M, Oro AE. Sebastiano V, et al. Sci Transl Med. 2014 Nov 26;6(264):264ra163. doi: 10.1126/scitranslmed.3009540. Sci Transl Med. 2014. PMID: 25429056 Free PMC article.