Use of Aptamers to deliver therapeutic genetic sequences in muscle

Abstract

There are several types of muscular dystrophy which have as a common characteristic muscle weakness and wasting. Antisense oligonucleotide approaches hold promise for the development of rational therapeutic approaches against muscular dystrophy. There is need to improve the delivery of such oligonucleotides in a tissue such as the muscle which is highly abundant in the human body. During the presentation, I am describing the identification and use of aptamers to enhance delivery in muscle.