genetically modified, non-self replicating Adeno-Associated Virus serotype 9 expressing shRNA to knock down mutant PABP1 as well as a codon optimized, shRNA-insensitive, wildtype PABN1

	genetically modified, non-self replicating Adeno-Associated Virus serotype 9 expressing shRNA to knock down mutant PABP1 as well as a codon optimized, shRNA-insensitive, wildtype PABN1
Trade Name
Orphan Indication	Oculopharyngeal muscular dystrophy
USA Market Approval	USA
USA Designation Date	2018-01-08 00:00:00
Sponsor	Benitec Biopharma;99 Mount Street, Suite 1201;North Sydney