RNA-targeted drugs for neuromuscular diseases
نویسندگان
چکیده
Progress with antisense oligonucleotide therapies opens a path for future development
منابع مشابه
RNA nanotechnology breakthrough for targeted release of RNA-based drugs using cell-based aptamers
Nucleic acids play different roles besides storing information and proteins coding. For example, single-stranded nucleic acids can fold into complicated structures with capability of molecular detection, catalyzing bioreactions and therapy. The development of RNA-based therapies has been rapidly progressed in the recent years. RNA aptamers are biomolecules with a size of 10 to 50 nm that can be...
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Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing is engineered to yield genetic correction, is a promising novel therapy for genetic diseases of muscle and nerve-the prototypical example being Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most common childhood genetic disease, affecting one in 3500 newborn boys, causing progressive muscle ...
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ژورنال
عنوان ژورنال: Science
سال: 2021
ISSN: ['2797-0744', '2797-1031']
DOI: https://doi.org/10.1126/science.aba4515