| 摘要 |
Disclosed are oligonucleotides which target and hybridize to nucleic acid molecules encoding A TXNJ, leading to reduced expression of ATXN3. Reduction in the expression of aberrant ATXN3 is beneficial for the treatment of certain medical disorders, such as spinocerebellar ataxia 3. In particular embodiments, modulating the expression of an aberrant A TXN3 allele or transcript, for example, restores normal function of, for example, Purkinje cells or spinal cord neurons. The oligonucleotides of the present invention and the methods of using such oligonucleotides to modulate the expression of aberrant or expanded A TXN3 provide a means of improving the survival and morbidity associated with, or even curing, expression of an aberrant A TXN3 allele or transcript such as, for example, spinocerebellar ataxia-type 3 (SCA3). |
