RNA therapeutics - Wikipedia
The main types of RNA therapeutics are those based on messenger RNA (mRNA), antisense RNA (asRNA), RNA interference (RNAi), and RNA aptamers. Of the four types, mRNA-based therapy is the only type which is based on triggering synthesis of proteins within cells, making it particularly useful in vaccine development. [3]
Taking RNAi from interesting science to impactful new treatments
2024年5月13日 · Alnylam Pharmaceuticals is translating the promise of RNA interference (RNAi) research into a new class of powerful, gene-based therapies. In this rendering, the green strand is the targeted mRNA, and the white object is the RNA-induced silencing complex (RISC) that can prevent the expression of the target mRNA’s proteins.
Advances in the delivery of RNA therapeutics: from concept to …
2017年3月6日 · In this review, we discuss the challenges for clinical translation of RNA-based therapeutics, with an emphasis on recent advances in delivery technologies, and present an overview of the applications of RNA-based drugs for modulation of gene/protein expression and genome editing that are currently being investigated both in the laboratory as ...
RNA therapeutics on the rise - Nature
2020年4月27日 · RNA therapeutics have demonstrated most success in the treatment of rare diseases, especially neurological and hepatic diseases. Of the 21 late-stage RNA therapeutics, 18 have orphan status...
RNA therapeutics: updates and future potential
2022年9月8日 · In this review, the general concepts of five classes of RNA-based therapeutics, including RNA interference-based therapies, antisense oligonucleotides, small activating RNA therapies, circular RNA therapies, and messenger RNA-based therapeutics, will be discussed.
The Limitless Future of RNA Therapeutics - Frontiers
2021年3月17日 · RNA therapeutics comprise a rapidly expanding category of drugs that will change the standard of care for many diseases and actualize personalized medicine. These drugs are cost effective, relatively simple to manufacture, …
RNA therapies explained - Nature
2019年10月16日 · Most RNA therapies can be sorted into one of three broad categories: those that target nucleic acids (either DNA or RNA), those that target proteins, and those that encode proteins.
THE SOCIETY FOR RNA THERAPEUTICS - Home
The SRT is a global network to support translational research and development of RNA therapeutics; establish standards for RNA manufacturing; create guidelines for clinical trials; facilitate public-private regulatory partnerships; to promote accessibility of RNA therapeutics for all patients; to offer public and professional education; to ...
RNA therapeutics : the evolving landscape of RNA therapeutics
Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies.
key shortcoming: RNA, in its usual linear form, is short-lived. Within hours, enzymes in cells descend on the molecule, chewing it to pieces. RNA’s fleeting nature isn’t a big problem