MORPHOLINO TARGETING DUX4 FOR TREATING FSHD

• Main Authors: MULTHAUP, MEGAN, KYBA, MICHAEL
• Format: Patent
• Language: English; French
• Published: 15.08.2013
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

A composition and method is provided that comprises a morpholino oligonucleotide capable of inhibiting DUX4 expression. The morpholino oligonucleotide can provide a therapeutic effect in individuals with facioscapulohumeral muscular dystrophy. Further, the composition and method may comprise a dendr...