Gene therapy of UBE3A transformed into Angelman's syndrome

摘要

The abstract of the invention introduces a new carrier, composition and treatment method of neural disorder characterized by UBE3A defect. UBE3A gene is encoded by E6-AP, a ligated ubelimina, which is found to be responsible for Angelman (as) syndrome. A unique feature of this gene is that it is impressed by the mother in a particular neurological way. In most cases, there is mutation or inhibition of UBE3A gene inheritance from the mother,Other cases are the result of unicellular crystallization or misuse of mother genes. A UBE3A protein structure is produced, increasing the sequence, allowing cell secretion and peripheral nerve cell absorption. The UBE3A vector can be used for gene therapy, providing functional E6-AP protein for the nervous system, and saving the pathology of the disease.