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BioWorld - Sunday, January 18, 2026
Home » Topics » Science » RNA

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Digital spine concept art
Neurology/Psychiatric

Nurexone reports new siRNA sequences with potential for spinal cord injury

Dec. 1, 2023
Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
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Heart illustration
Cardiovascular

Ethris and Heqet collaborate to develop RNA-based therapeutics for heart tissue regeneration

Nov. 13, 2023
Ethris GmbH and Heqet Therapeutics srl, a company spun out last year from King’s College London, have entered into a collaboration agreement to harness the potential of non-coding RNA (ncRNA) for heart tissue regeneration following acute myocardial infarction and in heart failure.
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3D rendering of a liposome containing RNA strand
Cancer

Scientists design a circular RNA against adenocarcinoma

Oct. 19, 2023
By Mar de Miguel
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.
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RNA illustration
Drug Design, Drug Delivery & Technologies

Deep Genomics announces AI foundation model for RNA

Sep. 28, 2023
Deep Genomics Inc. has introduced its artificial intelligence (AI) foundation model for RNA, BigRNA, which enables discovery of disease mechanisms and candidate therapeutics.
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3D rendered illustration of a synapse cross-section
Neurology/Psychiatric

Reduced RNA editing reveals mitochondrial dysfunction in schizophrenia

April 11, 2023
By Mar de Miguel
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.
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Endocrine/Metabolic

Korro Bio and Genevant Sciences partner on RNA editing therapeutic for alpha-1 antitrypsin deficiency

March 8, 2023
Korro Bio Inc. and Genevant Sciences Corp. have entered into an agreement to combine Korro's...
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Neurology/Psychiatric

Blocking nuclear export of ALS/FTD troublemaker helps neurons survive

March 3, 2023
By Anette Breindl
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.
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RNA strand
Drug Design, Drug Delivery & Technologies

Cas12a2 elicits abortive infection and can be repurposed as an RNA-guided RNA-targeting tool

Jan. 20, 2023
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.
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RNA illustration
Cancer

Esperovax and Ginkgo Bioworks partner on development of circular RNAs for therapeutic application

Jan. 9, 2023
Esperovax Inc. and Ginkgo Bioworks Inc. have established a partnership to develop circular RNAs (circRNAs) for a variety of therapeutic applications.
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3D rendering of Cas12a2 protein and DNA helix
Drug Design, Drug Delivery & Technologies

CRISPR-associated nuclease combines specific recognition with wanton destruction

Jan. 5, 2023
By Anette Breindl
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.
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