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A DNAzyme that targets the CAG repeat RNA in polyglutamine diseases
The invention is a synthetic single-stranded DNA molecule that functions as a specific RNA-cleaving DNA enzyme (or DNAzyme) as a potential therapeutic for the treatment of polyglutamine diseases. The binding arms of this synthetic DNAzyme are complementary to the CAG repeats found in the pathological expansion of CAG trinucleotide repeats in the translated...
Published: 3/12/2024
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Inventor(s): Nan Zhang, Tetsuo Ashizawa
Keywords(s):
Category(s): Clinical Area > Neurology, Research Area > Neurology and Neurosurgery, Technology Classifications > Therapeutic > Cell and Gene Therapies
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