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Long-term assessment of combined vitamin A and E treatment for the prevention of retinal degeneration in abetalipoproteinaemia and hypobetalipoproteinaemia patients

To draw RNA structures containing sequences predicted to form G4 structures, the ViennaRNA constraint option was employed, forcing the putative G4 tract to remain unpaired during secondary-structure prediction 59

At the same time, novel therapeutic paradigms are being applied in areas of high unmet medical need, including neurodegenerative disorders

Typically, AONs are 2030 nucleotide-long fragments of DNA or RNA, which, by binding to the exon/intron boundary or targeting intraexonic regions, can skip the particular exon(s), hide it from the splicing machinery, and restore the reading frame [75]

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