CELL-PENETRATING PEPTIDES: A DELIVERY SYSTEM FOR OLIGONUCLEOTIDES

オリゴヌクレオチドベースの医薬品の可能性に、ようやくその価値に見合った注目が集まっています。Bachemは新たなイノベーションをもたらし、弊社顧客、ひいては患者様のクオリティ・オブ・ライフの向上に貢献します。

添付のデリバリーシステムである細胞透過性ペプチド（CPP）についての記事をぜひご参照ください。

Oligonucleotides are very promising and versatile therapeutics for rare and genetic diseases. They act on the RNA level through different molecular pathways that we already have described in a precedent article. Because of their mechanism of action, this class of therapeutics has huge possibilities to treat diseases that were incurable before. The use of oligonucleotide therapeutics is well tolerated by the body, selective to their target, and with reduced secondary effects. Despites their huge potential and attractiveness as a treatment, there is a major drawback when dealing with oligonucleotides as drugs: their poor bioavailability and cellular uptake. These aspects are the main limitations for their application as therapeutics. To address these drawbacks, it seems of utmost importance to develop delivery systems that enable oligonucleotides to reach their targets. In a previous article on oligonucleotide conjugates, we have described the use of the trimeric GalNAc (N-acetylgalactosamine) molecule as a carrier of oligonucleotides to specific cells, the hepatocytes. Here, we discuss a delivery system that have been in vogue since almost 40 years, the cell-penetrating peptides (CPP). The first use of CPP, a poly L-lysine, has been described for the delivery of an anticancer drug in vitro and in vivo of mouse model1. It has opened the way for generation of CPP-drug conjugates and has potential as well for oligonucleotides2.
 

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