confocal microscopy imaging and small molecules for tri nucleotide repeats disorders
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Explanation:
Highlights
Highlights•
Highlights•Up to 34 drug candidates identified in the last few years.
Highlights•Up to 34 drug candidates identified in the last few years.•
Highlights•Up to 34 drug candidates identified in the last few years.•Drug screening methods available at all levels of complexity (computational to in vivo).
Highlights•Up to 34 drug candidates identified in the last few years.•Drug screening methods available at all levels of complexity (computational to in vivo).•
Highlights•Up to 34 drug candidates identified in the last few years.•Drug screening methods available at all levels of complexity (computational to in vivo).•Translational research has gained momentum.
Highlights•Up to 34 drug candidates identified in the last few years.•Drug screening methods available at all levels of complexity (computational to in vivo).•Translational research has gained momentum.Myotonic dystrophy type 1 (DM1) is a rare multisystemic neuromuscular disorder caused by expansion of CTG trinucleotide repeats in the noncoding region of the DMPK gene. Mutant DMPK transcripts are toxic and alter gene expression at several levels. Chiefly, the secondary structure formed by CUGs has a strong propensity to capture and retain proteins, like those of the muscleblind-like (MBNL) family. Sequestered MBNL proteins cannot then fulfill their normal functions. Many therapeutic approaches have been explored to reverse these pathological
Answer:
RNA trinucleotide repeat expansions cause or contribute to various neurological disorders (5) including Fragile