Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA molecules, 20-25 nucleotides in length, that play a variety of roles in biology. Most notably, siRNA is involved in the RNA interference (RNAi) pathway, where it interferes with the expression of a specific gene. In addition to their role in the RNAi pathway, siRNAs also act in RNAi-related pathways, e.g., as an antiviral mechanism or in shaping the chromatin structure of a genome; the complexity of these pathways is only now being elucidated.
invIOs GmbH (“invIOs”), a privately held biotechnology company developing innovative cancer immunotherapies, today announced the publication of new clinical data with its autologous PBMC (peripheral blood mononuclear cell) therapy APN401 in the peer-reviewed journal, Molecular Therapy Oncology.
Undecimal Bio, a platform biotechnology company advancing targeted delivery of siRNA therapeutics, today announced an oversubscribed $18 million USD seed financing.
Solid tumors are not merely collections of malignant cells but complex ecosystems comprising tumor cells, stromal cells, and the extracellular matrix (ECM).
Researchers at The University of Texas at Austin have teamed up with pharmaceutical leader Eli Lilly and Company to uncover new information about mRNA lipid nanoparticles (LNPs), the technology behind COVID-19 vaccines and other important treatments, and how to make it more effective.
A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.
Scientists from Nanyang Technological University, Singapore (NTU Singapore) have developed microscopic peptide droplets that deliver large amounts of gene-silencing molecules into cancer and immune cells, reducing the growth of hard-to-treat colorectal tumours by about 67 per cent in mice.
Several cannabinoids reduced chemotherapy- and radiation-induced apoptosis in glioblastoma cell models, with effects linked to reduced apoptotic priming, increased pro-survival signaling, and G1 cell-cycle arrest. The findings raise concern that cannabinoid exposure could interfere with some cancer treatments, although the results are limited to laboratory models and require in vivo and clinical validation.
Mega-scale exome sequencing of 1,032,116 people identified 59 independent genes linked to the TG:HDL ratio, including 44 rare-coding associations not detected in the previous largest analysis. Ultra-rare FNIP1 loss-of-function variants were associated with favorable fat distribution, lower liver fat and glycemia, and around 60% lower odds of a composite cardiometabolic disease outcome.
A recent review explores the rapidly expanding field of ferroptosis-based therapy for prostate cancer, presenting ferroptosis as a promising strategy to overcome resistance in advanced and castration-resistant prostate cancer (CRPC).
Cancer cells survive by hiding from the immune system's surveillance. A KAIST research team has developed a new anticancer platform that makes cancer cells send out their own danger signal—prompting immune cells to attack—while simultaneously delivering gene therapy.
RNA interference is a natural mechanism for living cells to control whether specific genes are being used or not.
Researchers at the University of Arizona R. Ken Coit College of Pharmacy have developed a new strategy that helps the immune system recognize and attack lung cancer tumors more effectively.
An experimental study in Science Advances shows that statins, primarily fluvastatin in these experiments, may promote low-level muscle myopathy by reducing isoprenoid-dependent protein prenylation rather than by cholesterol lowering. In primed muscle cells and mouse models, impaired YAP signaling, reduced glycolysis, NLRP3/caspase-1 activation, and nuclear FOXO accumulation increased atrogin-1, reduced protein synthesis, and weakened muscle function.
RNA therapeutics have emerged as one of the most promising new classes of medicines. Eight small interfering RNA (siRNA) drugs have already been approved worldwide for the treatment of genetic diseases, yet scientists have not fully understood one of the most fundamental steps underlying their function: how Argonaute, the core protein responsible for gene silencing, becomes activated.
While scientists have known for over two decades that all cells use a strategy called RNA interference to regulate gene expression, a new study is the first to describe how a specific protein manages the step-by-step process of assembling the molecular complex that performs the regulatory job.
National University of Singapore Researchers at the National University of Singapore (NUS) have developed a high-throughput method to identify gold nanoparticles capable of delivering therapies directly to mitochondria (the energy centres inside cancer cells).
Long-acting RNA interference therapy for hypertension could shift blood pressure control from daily pill-taking to scheduled, system-led protection. But the authors warn that this “vaccine-like” model must preserve lifestyle support, monitoring, and follow-up to avoid clinical disengagement.
A quiet revolution is underway in modern medicine: Drug development is aiming to move from managing disease to correcting it through RNA and gene-editing therapies.
Cancer stem cells (CSCs), a critical subpopulation within tumors, drive cancer initiation, progression, metastasis, relapse, and resistance to therapy due to their innate capacity for self-renewal and differentiation.
The tumor begins before birth. Somewhere in the developing fetus, neural crest cells that should have matured into adrenal tissue or sympathetic ganglia take a wrong turn, and a child is born harboring a malignancy that may not declare itself for months.
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