OSBORN, Mark, J.,TOLAR, Jakub,BLAZAR, Bruce,VOYTAS, Daniel, F.
申请号：
HK16107060.2
公开号：
HK1219021A
申请日:
2016.06.20
申请国别(地区):
HK
年份:
2017
代理人:
摘要：
The invention is directed to transcription activator-like effector nuclease (TALEN)-mediated DNA editing of disease-causing mutations in the context of the human genome and human cells to treat patients with compromised genetic disorders. Epidermolysis bullosa (EB) is a group of genetic conditions that cause the skin to be very fragile and to blister easily. Blisters and skin erosions form in response to minor injury or friction, such as rubbing or scratching. Recessive dystrophic epidermolysis bullosa (RDEB), the most severe and classical form of the disease, is characterized by extensive blistering and scarring of the skin and mucosal membranes.
