First mRNA Therapy for ISG15 Deficiency Developed

Researchers developed the first experimental mRNA-based therapy for ISG15 deficiency, a rare genetic mutation providing near-universal viral immunity, as reported on September 9, 2025.

Illumina AI Tool for Rare Diseases

11. syyskuuta 2025 Raportoinut AI

Illumina introduced an AI tool aiding in the diagnosis of rare diseases. This innovation enhances genetic analysis for complex conditions. It was highlighted in biotech news updates.

CRISPR Tool Targets Neuron Gene Repair

15. syyskuuta 2025 Raportoinut AI

Stanford researchers have created a CRISPR-based technology to target RNA in neurons, enabling repair processes for genetic diseases. The method addresses key challenges in Cas9 delivery and DNA repair. It reveals unique genome editing rules in neurons, paving the way for targeted therapies.

Study identifies potential genetic subtypes of autism

02. lokakuuta 2025 Raportoinut AI

Researchers have proposed that autism spectrum disorder may consist of genetically distinct subtypes, based on an analysis of large-scale genetic data. This finding could lead to more personalized approaches to diagnosis and treatment. The study highlights variations in genetic markers among individuals with autism.

DNA scar catalog opens paths to overcome cancer drug resistances

Researchers at Spain's National Cancer Research Centre (CNIO) have developed a comprehensive catalog of mutational 'scars' in DNA caused by cancer. This breakthrough, published in Nature, could help bypass resistances to treatments like PARP inhibitors. The study analyzes thousands of tumor samples to identify key genetic patterns.