Abstract: The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.
Type:
Application
Filed:
January 14, 2010
Publication date:
December 13, 2012
Applicant:
Philadelphia Health and Education Corp. d/b/a Drexel University College of Medicine
Inventors:
Gordon J. Lutz, Melanie K. Tallent, Nicole Michele Lykens
Abstract: This invention relates to drug delivery and specifically to the preparation and use of functionalized carriers such as nanopolymers and nanovesicles for improved delivery of nucleic acid agents (NAA) to tissues and cells. These compounds have broad applicability for treating numerous diseases and disorders, including neurodegenerative and neuromuscular disorders. The concept encompasses preferably polymeric carriers for delivery of a class of oligonucleotides that modulate RNA splicing.
Type:
Application
Filed:
January 2, 2007
Publication date:
January 8, 2009
Applicant:
Philadelphia Health & Education Corp., D/B/A/ Drexel University of College of Medicine
Inventors:
Gordon John Lutz, Margaret Alison Wheatley