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NeuBase Therapeutics

NeuBase has developed a modular antisense technology platform with the capability to address any rare genetic disease caused by mutant proteins with a single, cohesive approach. NeuBase's platform improves upon gene silencing therapies by combining the advantages of synthetic approaches with the precision of antisense technologies. NeuBase plans to use its unparalleled antisense platform to address both repeat expansion disorders, with an initial focus on Huntington's Disease and Myotonic Dystrophy, and dominant genetic disorders


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Antisense Oligonucleotides Make Sense

  
Antisense oligonucleotides (ASOs) are short (about 12 to 25 nucleotides long), synthetic single strands of DNA or RNA that are complementary to a chosen sequence. They can alter RNA and reduce, restore, or modify protein expression. ASOs interact with proteins on the surface of cells and enter the cytoplasm. Then …

Top Companies At The Forefront Of RNA Therapeutics Development

  
Alongside the topic of gene editing technologies that keeps making headlines these days, there is also a wave of breakthroughs in the field of RNA-targeting medicines, primarily falling into the following two categories: antisense oligonucleotides (ASO), and RNA interference (RNAi) technologies. (another promising approach, dealing with editing RNA itself by …