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Presentation Information

Session Details

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Program Type

A small molecule targeting UGGAA pentanucleotide repeats that cause spinocerebellar ataxia type 31

We focus on translational research related to mRNA, including therapeutics development for infectious and neurodegenerative diseases.  mRNA vaccines are now being rapidly and effectively deployed in the context of the COVID-19 pandemic.  Their design, synthesis, optimization, and formulation will be discussed, e.g., how to avoid immune rejection of the foreign nucleic acid. mRNA can also serve as the target for antisense-oligonucleotide (ASO) and small-molecule drugs.  ASOs can be designed to trigger degradation of the mRNA target or to alter how the mRNA is processed from its precursor; ‘Spinraza', e.g., modulates alternative splicing of SMN2 pre-mRNA and has been used to treat &gt;11,000 spinal muscular atrophy (SMA) patients worldwide.  A small-molecule drug, ‘Evrysdi', was recently approved to target the same splicing event in SMA.  Small molecules can be designed to selectively destroy target mRNAs or to alter their processing.  Both platforms will be discussed for various therapeutic applications.

日本分子生物学会年会

The 44th Annual Meeting of the Molecular Biology Society of Japan

The 44th Annual Meeting of the Molecular Biology Society of Japan

[1AS-01-5]A small molecule targeting UGGAA pentanucleotide repeats that cause spinocerebellar ataxia type 31

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