Ex‐vivo gene modification therapy for genetic skin diseases – recent advances in gene modification technologies and delivery

xlomafota13 shared this article with you from Inoreader Ex‐vivo gene modification therapy for genetic skin diseases – recent advances in gene modification technologies and delivery Via Experimental Dermatology Abstract Genetic skin diseases, also known as genodermatoses, are inherited disorders affecting skin and constitute a large and heterogeneous group of diseases. While genodermatoses are rare with the prevalence rate of less than 1 in 50,000 – 200,000, they frequently occur at birth or early in life and are generally chronic, severe, and could be life‐threatening. The quality of life of patients and their families are severely compromised by the negative psychosocial impact of disease, physical manifestations, and the lack or loss of autonomy. Currently, there are no curative treatments for these conditions. Ex‐vivo gene modification therapy that involves modification or correction of mutant genes in patients' cells in‐vitro and then transplanted back to patients to restore functional gene expression has being developed for genodermatoses. In this review, the ex‐vivo gene modification therapy strategies for genodermatoses are reviewed, focusing on current ad vances in gene modification and correction in patients' cells and delivery of genetically modified cells to patients with discussions on gene therapy trials which have been performed in this area. View on the web Inoreader. Take back control of your news feed. Follow us on Twitter and Facebook.