Antisense to c-myc for treatment of polycystic kidney disease
View Patent ↗A method of treating polycystic kidney disease by administering an oligonucleotide antisense to c-myc is described. The antisense oligonucleotide is preferably a morpholino oligonucleotide.
1. A method of treating polycystic kidney disease (PKD) in a human or murine subject, comprising:
administering to the subject a morpholino-based antisense oligomer having uncharged, phosphorous-containing intersubunit linkages, where the antisense oligomer contains the sequence identified as SEQ ID NO:1.
2. The method of claim 1 , where the intersubunit linkages of said morpholino-based antisense oligomer are uncharged, phosphorous-containing linkages one to three atoms long, joining the morpholino nitrogen of one subunit to the 5′ exocyclic carbon of an adjacent subunit.
3. The method of claim 2 , where said linkages include the phophorodiamidate linkage of structure II, where X═NH 2 , Y═O, and Z=O, and Pi and P j are purine or pyrimidine base-pairing moieties effective to bind, by base-specific hydrogen bonding, to a base in a polynucleotide.
4. The method according to claim 1 , where said administering takes place by any one of an oral route, a subcutaneous route, an intranasal route, a transdermal route or an intravenous route.
5. The method according to claim 4 , where said administering takes place by an oral route.
6. The method of claim 1 , where said subject is a human subject.
7. The method according to claim 1 , where the antisense oligomer is administered in a manner effective to reduce or eliminate a detectable symptom of PKD in the subject.