Long-term skin regeneration from a gene-targeted human epidermal stem cell clone

Molecular Therapy

Volumn 22, Issue 11, 2014, Pages 1878-1880

  Duarte, Blanca ^a,b   Miselli, Francesca ^c   Murillas, Rodolfo ^a,b   Espinosa Hevia, Luis ^d   Cigudosa, Juan Cruz ^d   Recchia, Alessandra ^c   Río, Marcela Del ^b,e   Larcher, Fernando ^a,b,e  

^a Cutaneous Diseases Modeling Unit   (Spain)

^b UAM)   (Spain)

^c UNIVERSITY OF MODENA AND REGGIO EMILIA   (Italy)

^d Structural Biology and Biocomputing Programme   (Spain)

^e UNIVERSIDAD CARLOS III DE MADRID   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

4 (1 AMINOETHYL) N (4 PYRIDYL)CYCLOHEXANECARBOXAMIDE; ENHANCED GREEN FLUORESCENT PROTEIN; MBS85 PROTEIN, HUMAN; PHOSPHOPROTEIN PHOSPHATASE 1;

AAVS1 GENE; BIOENGINEERING; CELL CLONE; CULTURE MEDIUM; DNA DETERMINATION; DNA ISOLATION; EPIDERMAL STEM CELL; FLOW CYTOMETRY; FLUORESCENCE ANALYSIS; GENE; GENE LOCUS; HUMAN; KERATINOCYTE; LETTER; NONHUMAN; POLYMERASE CHAIN REACTION; PROTEIN EXPRESSION; SKIN BIOPSY; SKIN REGENERATION; SOUTHERN BLOTTING; STEM CELL EXPANSION; TISSUE REGENERATION; VIRAL GENE DELIVERY SYSTEM; ANIMAL; CYTOLOGY; DEPENDOPARVOVIRUS; EPIDERMIS; GENE THERAPY; GENE VECTOR; GENETICS; METABOLISM; MOUSE; PROCEDURES; SKIN DISEASES, GENETIC; SKIN TRANSPLANTATION; STEM CELL; VIROLOGY;

ANIMALS; DEPENDOVIRUS; EPIDERMIS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; KERATINOCYTES; MICE; PROTEIN PHOSPHATASE 1; SKIN DISEASES, GENETIC; SKIN TRANSPLANTATION; STEM CELLS;

EID: 84964314240     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.187     Document Type: Letter

References (13)

1. Uitto, J (2012). Rare heritable skin diseases: Targets for regenerative medicine. J Invest Dermatol 132: 2485-2488.
2. Mavilio, F, Pellegrini, G, Ferrari, S, Di Nunzio, F, Di Iorio, E, Recchia, A et al. (2006). Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 12: 1397-1402.
3. Osborn, MJ, Starker, CG, McElroy, AN, Webber, BR, Riddle, MJ, Xia, L et al. (2013). TALEN-based gene correction for epidermolysis bullosa. Mol Ther 21: 1151-1159.
4. Coluccio, A, Miselli, F, Lombardo, A, Marconi, A, Malagoli Tagliazucchi, G, Gonçalves, MA et al. (2013). Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol Ther 21: 1695-1704.
5. Nanba, D, Matsushita, N, Toki, F and Higashiyama, S (2013). Efficient expansion of human keratinocyte stem/progenitor cells carrying a transgene with lentiviral vector. Stem Cell Res Ther 4: 127.
6. Zhou, Q, Duan, H, Wang, Y, Qu, M, Yang, L and Xie, L (2013). ROCK inhibitor Y-27632 increases the cloning efficiency of limbal stem/progenitor cells by improving their adherence and ROS-scavenging capacity. Tissue Eng Part C Methods 19: 531-537.
7. Mathor, MB, Ferrari, G, Dellambra, E, Cilli, M, Mavilio, F, Cancedda, R et al. (1996). Clonal analysis of stably transduced human epidermal stem cells in culture. Proc Natl Acad Sci USA 93: 10371-10376.
8. Barrandon, Y and Green, H (1987). Three clonal types of keratinocyte with different capacities for multiplication. Proc Natl Acad Sci USA 84: 2302- 2306.
9. Melo, SP, Lisowski, L, Bashkirova, E, Zhen, HH, Chu, K, Keene, DR et al. (2014). Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. Mol Ther 22: 725-733.
10. García, M, Llames, S, García, E, Meana, A, Cuadrado, N, Recasens, M et al. (2010). In vivo assessment of acute UVB responses in normal and xeroderma pigmentosum (XP-C) skin-humanized mouse models. Am J Pathol 177: 865-872.
11. Di, WL, Larcher, F, Semenova, E, Talbot, GE, Harper, JI, Del Rio, M et al. (2011). Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts. Mol Ther 19: 408-416.
12. Aufenvenne, K, Rice, RH, Hausser, I, Oji, V, Hennies, HC, Rio, MD et al. (2012). Long-term faithful recapitulation of transglutaminase 1-deficient lamellar ichthyosis in a skin-humanized mouse model, and insights from proteomic studies. J Invest Dermatol 132: 1918-1921.
13. Larcher, F, Dellambra, E, Rico, L, Bondanza, S, Murillas, R, Cattoglio, C et al. (2007). Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy. Mol Ther 15: 1670-1676.