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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]
An mRNA vaccine is a type of vaccine that uses a copy of a molecule called messenger RNA (mRNA) to produce an immune response. [1] The vaccine delivers molecules of antigen -encoding mRNA into cells , which use the designed mRNA as a blueprint to build foreign protein that would normally be produced by a pathogen (such as a virus ) or by a ...
A 5' cap (also termed an RNA cap, an RNA 7-methylguanosine cap, or an RNA m 7 G cap) is a modified guanine nucleotide that has been added to the "front" or 5' end of a eukaryotic messenger RNA shortly after the start of transcription. The 5' cap consists of a terminal 7-methylguanosine residue that is linked through a 5'-5'-triphosphate bond to ...
The modified gene therapy strategy of antisense IGF-I RNA (NIH n˚ 1602) [168] using antisense / triple helix anti-IGF-I approach was registered in 2002, by Wiley gene therapy clinical trial - n˚ 635 and 636.
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
A nucleoside-modified messenger RNA (modRNA) is a synthetic messenger RNA (mRNA) in which some nucleosides are replaced by other naturally modified nucleosides or by synthetic nucleoside analogues. [ 1 ] modRNA is used to induce the production of a desired protein in certain cells.
Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available); Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma [1]
The first use of RNA for vaccination purposes was described in 1993 by Frédéric Martinon, Pierre Meulien and colleagues [10] [11] and in 1994 by X. Zhou, Peter Liljeström, and colleagues in mice. [12] [11] Martinon demonstrated that a cellular immune response was induced by vaccination with an RNA vaccine. [11]