GoKawiil
Tech News
A man with a slow-progressing, genetically rare form of ALS became the first person to receive an antisense oligonucleotide therapy designed specifically to target his causal mutation. A year later, he reported improved symptoms and remains able to work as a physician, according to results published in Med.
Scientists are engineering particle-based delivery systems intended to ferry large molecules such as proteins and messenger RNA past the blood-brain barrier, a membrane that normally blocks such compounds from reaching brain tissue. Small chemicals like paracetamol already pass through naturally, but bigger therapeutic molecules cannot, limiting treatment options for brain disorders.
Researchers modified the tethered Ribo-T ribosome system in E. coli by appending open reading frames for GFP, DFR and HiBiT directly onto the 3′ end of 16S rRNA, creating 'Ribo-TM' constructs. Gel electrophoresis and RT-PCR confirmed the fused mRNA-rRNA molecules were intact and correctly assembled inside functioning ribosomes, and growth assays with mutant ribosomal subunits helped verify that translation activity depended on a functional ribosome.
Scientists engineered synthetic protein assemblies from the bottom up that can package and transport RNA molecules into human cells, using standard molecular cloning and transfection techniques in HEK293T cell lines. They validated the approach with a split-luciferase reporter system to confirm successful RNA delivery and functional expression inside cells.
Researchers describe a workflow that synthesizes and in-vitro-tests large pools of tRNA variants, including E. coli, M. alvus and M. mazei isoacceptors, using microchip-derived DNA oligo pools, T7-driven transcription, and cell-free translation systems. The method allows rapid amplification, refolding, purification and aminoacylation testing of tRNAs to evaluate their function outside living cells.
Harvard researchers studying ribosomal RNA in the California two-spot octopus discovered an unprecedented gap that splits a normally single RNA fragment into two pieces, a feature not seen in any other animal studied so far. This structural quirk, published in Current Biology, appears to help ribosomes assemble proteins with unusual precision, and so far turns up only in octopus lineages with enlarged nervous systems capable of complex behavior.