UBE3A for treating angel syndrome

Abstract

The angel syndrome is a hereditary neurological disorder, and is characterized by development retardation, intellectual disability, serious speech disorder and exercise and balance problems. Provided herein are polynucleotides, vectors, polypeptides, cells, compositions, kits and methods for treating angel syndrome.

Description

[0001] Cross References to Related Applications [0002] This application claims priority under 35 U.S.C. §119(e) to U.S. Provisional Application Nos. 62 / 890,364 and 62 / 945,062, filed August 22, 2019, and December 6, 2020, respectively, the contents of each of which are adopted by References are incorporated in this application in their entirety. Background technique [0003] Angelman syndrome (AS) is an inherited neurological disorder characterized by developmental delay, intellectual disability, severe speech impairment, and problems with movement and balance. Most patients have recurrent seizures and small head size. Most patients show developmental delay, and other common symptoms appear in early childhood. Children with AS often have a happy and excited demeanor. Other symptoms include hyperactivity, short attention span and fascination with water. People with Angelman syndrome become less aroused and sleep problems improve as patients age. However, these patients wi...

AI Technical Summary

Problems solved by technology

There are no effective therapies available for the treatment of AS

Images