Genome Editing

New software aims to help cell and gene therapy companies with manufacturing scaleup.

New insights into the genetic basis of a neonatal diabetes has uncovered a biological pathway that is fundamental to insulin production by pancreatic β cells, and which could boost research into new treatments for more common forms of diabetes. The international research team identified genetic changes in a cell trafficking gene, called YIPF5, in a number of neonatal diabetes patients. Harnessing stem cell and CRISPR technologies, the research showed how lack of, or mutations in the YIPF5 gene result in high levels of stress within the β cells, causing cell death.

Novel non-sequencing technique was designed to improve advanced therapy safety.

Vedere Bio recently became a wholly-owned subsidiary of Novartis after being acquired by the pharma giant for up to $280 million--$150 million upfront, up to $130 million in milestones—in a deal designed to bolster the buyer’s expanding presence in gene therapy

The goal of the center is to bring together industry, technology developers, academia, and regulatory agencies to help advance the development and manufacturing of cell and gene therapies.

In glaucoma, the optic nerve that connects the eye to the brain is progressively damaged, often in association with elevated pressure inside the eye. Researchers used gene therapy to regenerate damaged axons in the eye, in a discovery that could aid the development of new treatments for glaucoma, one of the leading causes of blindness worldwide.

In solving the long-standing mystery of the function of bacterial structures called retrons, genetic elements comprised of a reverse transcriptase and a non-coding RNA, a group has uncovered a new strategy used by bacteria to protect themselves against viral infection. The research revealed many new retrons that may, in the future, add to the genome-editing toolkit.

Researchers at the University of Washington, and at Microsoft, have developed a DNA-based molecular tagging system, called Porcupine, which they say could offer a cost effective alternative to bulky plastic or printed barcodes that are commonly used in retail or manufacturing. The new molecular tags are built from dehydrated strands of synthetic DNA, which can be programmed and read within seconds using a portable nanopore device. The developers claim their technology represents the first portable, end-to-end molecular tagging system that enables rapid, on-demand encoding and decoding at scale.

Applying CRISPR-Cas9 to repair a blindness-causing gene in a human embryo often eliminates an entire chromosome or a large section of it, report Columbia University scientists. These losses occur depending on how an induced double-strand break is repaired, suggesting that alternative approaches, such as base or prime editing, may be preferred in some applications.

Virtual reality may become a standard training method as more companies enter the advanced therapy market.