Lesion of inherited skin diseases and gene therapy

Skin disorders contain some congenital problems that cause lacking structure proteins in the basement membrane zone and show serious prognosis because of severe erosion and skin dysfunction on the whole body. In fact, about 500 different genes are now known in harbor mutations that illustrate the genetic ulcer in these situations. Since these disorders lead to a severe lesion, some therapeutic strategies have been appearing for them: 1. protein therapies, 2. gene therapies, and 3. cell therapies. The qualities of skin cause that it be desirable for gene therapy and researchers have focused on gene therapy for multiple dermal disorder. Gene therapy uses a vector to introduce genetic material into cells to alter gene expression, negating a pathological process. This can be accomplished with a variety of viral vectors or nonviral administrations. Ex vivo and in vivo gene therapy technique for dystrophic epidermolysis bullosa have been examined greatly, often in immunedeficient small animal models and lately in dogs. Lentiviruses, retroviruses, and nonviral transposon mediated gene- transfer methods have been used successfully for transduction of fibroblasts and keratinocytes in this disorder. Hence, meganucleases, zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) have been used extensively for genome editing in a different type of cell. Recently, targeted genome editing using CRISPRCas9 systems has quickly used from being a niche technology as the main procedure because of cost reduction for reaching a new level of efficiency and targeting. Although Promising results obtained through genome editing technique, confirmatory and final studies are required

Keywords:

Skin , Disorder , Gene therapy