Genome Editing

In a series of experiments using human cancer cell lines, scientists at Johns Hopkins Medicine say they have successfully used light as a trigger to make precise cuts in genomic material rapidly, using a molecular scalpel known as CRISPR, and observe how specialized cell proteins repair the exact spot where the gene was cut.

Using induced pluripotent stem cells and deleting a key gene, researchers have created natural killer cells with measurably stronger activity against a form of leukemia, both in vivo and in vitro. Deleting the gene removes a brake on IL-15 signaling, which improves NK cell activation and function. It also leads to metabolic reprogramming of the NK cells, which become more efficient at energy utilization. The new approach has other advantages. iPSCs provide a stable platform for gene modification, and NK cells can be used as allogeneic cells that do not need to be matched to individual patients.

CTX001 also shows effectiveness in a patient with sickle cell disease (SCD), according to results from two Phase I/II trials that are the first clinical studies of a gene-editing candidate sponsored by U.S. companies.

A team of scientists from the University of Toronto has utilized genome-wide CRISPR screens to investigate global changes in cancerous cells as they adapt to a shortfall of critical nutrients such as lipids, which make up the cell's outer membrane. When cancer cells are unable to make their own lipids, they gobble them up from their environment to ensure a steady supply of these essential building blocks, the study found. Lipids also serve as fuel and chemical signals for communication between cells, among other roles.

SWFT is designed to foster collaboration and coordination between research teams by predicting scheduling and occupancy conflicts, which allows team members to adjust their schedules in order to promote social distancing in the lab and office.

Researchers have succeeded in inhibiting the immune response induced by AAV antibodies using Imlifidase (IdeS), an enzyme that is able to degrade circulating IgG. This advance provides a potential solution to the limitations that exist due to pre-existing antibodies to AAV-based gene therapy, and may allow for repeated administration of gene therapy. The results open up new therapeutic prospects and the possibility of treating more patients.

Synthetic biology seeks to create adaptive, sustainable, and dynamic biomaterials, and candidates for such materials include spatially organized bacterial communities. But realizing such communities requires precision, robustness, and easy-to-implement technology. 3D bioprinters may not suffice, since they tend to lay down static patterns. A potential alternative is meniscus-driven fluidics, or MeniFluidics.

We all hear lots about the life sciences clusters around Boston and San Francisco. But many clusters across the nation deserve our attention too—not just the major hubs.

Researchers at the ChristianaCare’s Gene Editing Institute have discovered that a mutation unique to certain cancer tumors may serve as a potential signal for safely deploying CRISPR gene editing enzymes to disarm DNA that makes cancer cells resistant to treatment, without affecting healthy cells.

Gene therapy base editors were developed to treat a mouse model of recessive genetic deafness caused by a point mutation in the transmembrane channel-like 1 gene, Tmc1. This method, which is the first example of repairing a recessive gene mutation, restored hair cell sensory transduction and partial hearing in the mice. These results suggest that base editing might be effective for rescuing recessive genetic hearing loss.