First proof-of-concept in vivo data for RNA editing using endogenous ADAR enzymes in alpha-1 antitrypsin deficiency ADAR editing resulted in therapeutically meaningful restoration of circulating functional AAT protein Wave’s program in alpha-1 antitrypsin deficiency aims to correct the single base mutation in mRNA derived from the SERPINA1 Z allele, thereby addressing both lung and liver manifestations of the disease CAMBRIDGE, Mass., June 02, 2021 (GLOBE NEWSWIRE) Wave Life Sciences Ltd. (Na
March 10, 2021 Researchers found that RNA editing events can have large downstream effects that influence gene expression and subsequent phenotypic manifestations of that expression. RNA editing may be a previously underappreciated mechanism driving human diseases, according to an analysis published in
RNA editing is a cellular process in which the sequence in mature RNA molecules is enzymatically altered from the genomic sequence. Altered levels of RNA editing have been associated with cancers and various other neurological conditions.
The most common RNA editing event in animals is adenosine (A) to an inosine (I) RNA editing, in which adenosines are deaminated to inosines. This A-to-I RNA editing is mediated by members of the adenosine deaminase acting on RNA (ADAR) family.
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