GENE THERAPY FOR RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA USING GENETICALLY CORRECTED AUTOLOGOUS KERATINOCYTES

摘要

FIELD: biotechnology.;SUBSTANCE: invention relates to the field of medical biotechnology. Proposed is an application of a keratinocyte layer for treatment of recessive dystrophic Epidermolysis Bullosa (RDEB). A keratinocyte layer derived from a population of skin cells for healing wounds in a subject with recessive dystrophic Epidermolysis Bullosa. The skin cells are transduced with a virus containing a genetic construct. The construct encodes a functional collagen VII protein (COL7A1). A method for producing a keratinocyte layer.;EFFECT: invention allows to perform genetic correction of RDEB using an improved technique of local delivery and expression of functional and full-length wild-type human collagen YII in affected skin; efficient delivery and expression of COL7AI in keratinocytes allows to use the described cells in treatment of epidermolysis bullosa.;26 cl, 13 dwg, 3 tbl, 1 ex