PYC pyc therapeutics limited

Sarepta's FDA rejection, page-7

  1. 5,481 Posts.
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    Thanks Hottod. Im definately no scientist and still trying to understand the terminology. After recently reading the article section below i had assumed a carrier was neccesary for a PMO but dont know. I found the article interesting though. Not sure if it was posted before by others.

    "As PMOs do not efficiently enter cells on their own, the strategy of CPP conjugation has been utilized to improve cellular uptake and enhance their antisense activity [38]. Conjugation of CPP to PMO uses several methods, including maleimide linkage, disulfide linkage, click chemistry, or amide linkage [39] and enhances the PMO pharmacokinetic profile, biodistribution, and stability [40]. The most promising results to date have been with arginine-rich peptides that contain aminohexanoic acid and beta-alanine spacers to improve delivery of splice-switching oligonucleotides (SSOs) "

    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6027240/
 
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