The mRNA technology used in vaccines against viral infections could also be developed for cancer therapies. A group of scientists has designed a circular RNA (circRNA) encapsulated in lipid nanoparticles (LNPs) that acts in the mitochondria of tumor cells through the protein gasdermin-D (GSDMD) and reduces adenocarcinoma. The work was published on Oct. 16, 2023, in Nature Cancer.
Deep Genomics Inc. has introduced its artificial intelligence (AI) foundation model for RNA, BigRNA, which enables discovery of disease mechanisms and candidate therapeutics.
RNA editing in schizophrenia (SCZ)-associated genes was decreased in postmortem brains of individuals of European descent, according to a study from the University of California, Los Angeles (UCLA). The scientists obtained the RNA editome from SCZ brains to detect the sequence changes in their RNA and observed hypoediting in noncoding regions related to mitochondrial function, such as the mitofusin-1 (MFN1) gene.
Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
Bacterial abortive infection is a defense mechanism by which an infected bacterial cell enters dormancy or dies to limit phage replication and protect the clonal population. Recent studies observed that CRISPR RNA-guided adaptive immune systems that target RNA also cause abortive-infection phenotypes by activating indiscriminate nucleases.
CRISPR, or clustered regularly interspaced palindromic repeats, is transforming biomedical research, and making rapid inroads into the clinic, with its ability to easily target specific DNA and RNA sequences. CRISPR itself is made of RNA. It recognizes target sequences and delivers CRISPR-associated (Cas) proteins, nucleases that cut the target sequence. In two papers published online in Nature on Jan. 4, 2023, researchers have demonstrated that a recently discovered type of Cas protein, Cas12a2, can degrade double-stranded DNA when its associated CRISPR guide RNA recognizes its target sequence.
Ice, juice, the exact measure of liquor, a few drops of Angostura... What goes into a good New Year’s Eve cocktail? According to researchers working on vaccines for the most elusive viruses, it will be time soon to toast next-generation vaccines. If 2020 was the year of the COVID-19 pandemic, and in 2021 the year of mRNA vaccinations, 2022 brought polyvalent designs of antigens, evaluated highly neutralizing antibodies, and fine-tuned mRNA technology against SARS-CoV-2, HIV and the flu.
Wave Life Sciences Ltd. and GSK plc have entered into a strategic collaboration to advance oligonucleotide therapeutics, including Wave's preclinical RNA editing program targeting α1-antitrypsin deficiency (AATD), WVE-006. The discovery collaboration has an initial 4-year research term. The first part of the arrangement is a discovery collaboration that enables GSK to advance up to eight programs and Wave to advance up to three programs, leveraging Wave's PRISM oligonucleotide platform and GSK's expertise in genetics and genomics.