CRISPR and Genome Editing – News and Features

Researchers developed a dual-AAV gene therapy for Usher syndrome type 1F, restoring hearing and balance in mice. The approach effectively delivered the large PCDH15 gene and showed promise in retinal organoids and nonhuman primates.

MIT researchers reveal how exercise stimulates neuron growth via biochemical and mechanical effects of muscle activity. Myokines and mechanical stretching enhance nerve growth and maturity.

The Discovery Spark program aims to help upcoming biotechnology and healthcare companies grow. In this article, we take a closer look at some of the innovative technologies of this year's Discovery Spark cohort.

A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.

A study reveals distinct brain circuits that psychedelics activate to reduce anxiety without causing hallucinations in mice. By isolating the anti-anxiety effects from hallucinogenic responses, researchers hope to develop safer, targeted treatments.

Northeastern University researchers introduce MECHS, a bioplastic that dissolves in water and decomposes in compost. Made from engineered E. coli bacteria and a fiber matrix, it mimics plastic's strength and flexibility.

UW–Madison researchers used gene therapy to prevent hereditary spastic paraplegia (HSP) in a rat model, introducing a healthy version of the Trk-fused gene to compensate for the mutated one. This prevented the onset of HSP symptoms in rats.

ETH Zurich researchers used combined CRISPR techniques to reveal unknown cancer-related mutations in the EGFR gene, identifying variants that may cause cancer or confer drug resistance.

Scientists at Vilnius University have developed a novel gene silencing method using a type IV-A CRISPR system that silences genes without DNA cuts. This approach could lead to safer gene-editing tools.

Using CRISPR-Cas13, NYU researchers reveal that noncoding RNAs, previously deemed “junk,” are crucial for cell function and development. Their findings, published in Cell, identify nearly 800 essential noncoding RNAs.