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New therapy may reverse autism-related brain deficits

Researchers identified a promising new strategy for reversing autism-related brain deficits by targeting a specific glycine transporter. The therapy restored NMDA receptor function in mouse models and human brain organoids, improving behavioral abnormalities such as social interaction and repetitive behaviors.

SourceInstitute for Basic Science·JournalNature Communications·TypeExperimental study·DateJun 9, 2026

Short-circuiting pancreatic cancer

Researchers have discovered a complex regulatory circuit involving SRSF1, AURKA, and MYC that promotes aggressive pancreatic cancer progression. The circuit, which involves alternative splicing, can be targeted with an antisense oligonucleotide to reduce tumor cells' viability and trigger apoptosis.

SourceCold Spring Harbor Laboratory·JournalMolecular Cell·DateJan 8, 2026

New way to improve the efficacy of innovative RNA therapies

Researchers discovered that slowing down intracellular transport of RNA-based drugs increases their effectiveness in treating genetic diseases. The study identified key genes involved in endosomal transport and found that selectively switching off a specific gene can prolong ASO residence time, boosting therapeutic efficacy.

SourceUniversity of Basel·JournalNature Communications·DateJun 30, 2025

Chung-Ang University researchers unveil the biogenesis and role of transfer RNA fragments in cancer progression

Researchers at Chung-Ang University have identified a crucial role for specific tRNA fragments in cancer progression, revealing their ability to regulate gene expression and influence tumor growth. The study suggests that these fragments could serve as biomarkers for early-stage cancer detection and targets for therapeutic interventions.

SourceChung Ang University·JournalNature Communications·TypeExperimental study·DateDec 11, 2024

Scientists use microcellular drones to deliver lung cancer-killing drugs

Researchers successfully delivered anti-cancer ASO molecules to lung tumor sites using human red blood cells, demonstrating potent anti-cancer effects against NSCLC. The approach utilizes EGFR-targeting moieties to home in on cancerous cells, offering a potentially powerful treatment modality for personalized cancer medicine.

Antisense oligonucleotide treatment shows promise in treating Parkinson's disease progression

Researchers from Tokyo Medical and Dental University demonstrate a proof of concept for antisense nucleic acid therapy to prevent the spread of α-synuclein pathologies in synucleinopathies. The treatment, involving antisense oligonucleotides, effectively reduces Lewy pathology-like neuronal inclusion by over 90%.

SourceTokyo Medical and Dental University·JournalActa Neuropathologica Communications·DateJul 25, 2024

How cells boost gene expression

A research team from Göttingen University has discovered that antisense RNA (asRNA) plays a crucial role in cell transport, allowing cells to accelerate gene expression and produce proteins quickly in response to environmental stress or harm. This new understanding sheds light on the function of asRNAs and their potential link to disea...

SourceUniversity of Göttingen·JournalNature·TypeExperimental study·DateJun 24, 2024

Next-generation treatments hitch a ride into cancer cells

Researchers from Osaka University have discovered a way to deliver antisense oligonucleotides to their targets inside cancer cells by opening specific calcium permeable channels. The new compound, L687, promotes efficient uptake of ASO into cancer cells, suppressing target gene activity and enhancing ASO efficacy.

SourceOsaka University·JournalNucleic Acids Research·TypeExperimental study·DateApr 15, 2024

Scientists develop novel RNA- or DNA-based substances to protect plants from viruses

Researchers at Martin-Luther-Universität Halle-Wittenberg developed novel RNA- or DNA-based substances that reliably fight off viral infections in plants. The new approach uses antisense oligonucleotides to target specific viral RNA molecules, achieving an impressive up to 90% success rate against a common virus.

SourceMartin-Luther-Universität Halle-Wittenberg·JournalInternational Journal of Molecular Sciences·TypeExperimental study·DateFeb 29, 2024

Antisense therapy restores fragile X protein production in human cells

A novel antisense therapy has restored fragile X protein production in human cell samples, revealing aberrant alternative splicing of messenger RNA as a key factor in fragile X syndrome. This finding offers real hope for developing new treatments and improving the lives of individuals affected by the condition.

SourceUMass Chan Medical School·JournalProceedings of the National Academy of Sciences·TypeExperimental study·DateJul 5, 2023

Peanut studies reveal surprising truths about RNA and open the door to better understanding epigenetic mechanisms in plants

Scientists have discovered a new layer of regulation in plant-microbe interactions using peanut studies. An antisense long-noncoding RNA, DONE40, was found to bind to a protein involved in epigenetic control, suggesting a conserved function across plants and animals.

SourceAmerican Phytopathological Society·JournalMolecular Plant-Microbe Interactions·TypeExperimental study·DateJan 13, 2022

UD researchers discover novel 'gene toggles' in world's top food crop

University of Delaware researchers have discovered a new type of molecule called natural antisense microRNAs (nat-miRNAs) that can turn off genes in rice, which is the primary source of food for half the world's population. These novel molecules may help scientists locate similar gene regulators in other organisms, including humans.

SourceUniversity of Delaware·JournalProceedings of the National Academy of Sciences·DateApr 9, 2008

Scientists target microbe with sleeker antisense agent

Researchers at the University of Rochester have created a remarkably short antisense compound that targets Pneumocystis carinii, an opportunistic pathogen causing pneumonia in people with weakened immune systems. The breakthrough marks a step toward designing drugs that knock out vital sections of molecules essential for the microbe's ...

SourceUniversity of Rochester·JournalProceedings of the National Academy of Sciences·DateJun 25, 1999