CRISPR and Genome Editing – News and Features

St. Jude researchers developed a 3D atlas tracing brain connections to spinal V1 interneurons, crucial for movement. Using modified rabies virus and serial two-photon tomography, they mapped neural pathways for motor control.

University of Illinois researchers developed SPLICER, a gene editing tool enhancing exon skipping. Targeting Alzheimer’s-related genes in mice, SPLICER decreased amyloid-beta plaque precursors by 25%, outperforming CRISPR techniques.

The findings reveal that climate change and measures to control populations of tsetse flies, which carry the disease, may drive molecular changes in the organisms that cause sleeping sickness – which can be fatal if left untreated.

A chemogenetic tool could form the basis of a gene therapy for chronic pain by turning off pain receptors.

McGill researchers discovered that K884, an experimental drug, enhances muscle stem cell repair in Duchenne muscular dystrophy (DMD). Unlike gene therapies, K884 targets DMD-affected cells regardless of the mutation.

Cell and gene therapies are revolutionizing medicine. Looking to 2025, advancements in allogeneic therapies, streamlined autologous processes and broader applications promise increased accessibility and innovation in treating complex diseases.

The ACTIMOT method, developed at Helmholtz Institute, uses CRISPR-Cas9 to extract and amplify biosynthetic gene clusters in bacteria, unlocking hidden natural products. Researchers identified 39 new bioactive compounds.

In a new study, scientists combined strategies to deliver energy-disrupting gene therapy using nanoparticles manufactured to zero in only on cancer cells, with experiments showing the targeted therapy effective in mice.

The delicate balance between immune system rest and activation is crucial for our health. For T cells, this balance is controlled by a single protein.

This article explores the latest techniques and workflows for purification across biopharmaceuticals including vaccines, monoclonal antibody therapies and gene therapies.