Efficient Gene Editing with Cellular Precision

A new highly efficient and precise gene editing technology developed at UC Santa Barbara (see below) could have huge long-term potential.

"We can actually hit individual cells," says research leader Norbert Reich. "We can even hit parts of a cell so we could release the protein into only a part of the cell. But the main point is that we have the control over where and when this protein that's going to cut the DNA is going to be released."

"The importance of this is that current approaches to gene editing often result in the editing machinery being left in an active form in the targeted cell, with unknown long-term ramifications," added a researcher. "Our approach delivers the editing machinery in a transient fashion, and thus circumvents this problem."

New high-precision gene editing technology. Researchers at UC Santa Barbara have developed a new gene editing technique, estimated to be 100 to 1,000 times more efficient than current methods, which permits deciding exactly when and where to release gene editing proteins. The technique is based on Cre “molecular scissors,” described in a study published in Small. According to the scientists, the potential is massive, and ranges from basic research that determines the function and identification of genes to therapies that could fix cellular-level defects.

New drug shows 50 percent cure rate in lab mice with acute leukemia. Scientists at the Hebrew University of Jerusalem have developed a new biological drug with a cure rate of 50 percent for lab mice with acute leukemia. The new single molecule drug, described in a research paper published in Cell, attacks several leukemic proteins at once, making it difficult for the leukemia cells to activate other proteins that can evade the therapy. The drug also accomplishes the work of multiple separate drugs, and is able to eradicate leukemia stem cells.

Cancer immunotherapy method shows promising results in clinical trial. Combination immunotherapy shrank melanoma that has spread to the brain in more than half of the patients in a clinical trial reported in the New England Journal of Medicine and led by an investigator at The University of Texas MD Anderson Cancer Center. The patients were treated with ipilimumab, which blocks the CTLA-4 checkpoint on T cells, in combination with nivolumab, which inhibits activation of the PD1 checkpoint. Both checkpoints otherwise shut down T cells and thus block the anti-tumor immune response.

AI system detects small cancers in CT scans better than humans. Engineers at University of Central Florida have taught a computer how to detect tiny specks of lung cancer in CT scans, which radiologists often have a difficult time identifying. According to the scientists, the new Artificial Intelligence (AI) system, described in a paper accepted for the MICCAI 2018 conference, is about 95 percent accurate, compared to 65 percent when done by human eyes.

Molecules for infection treatment and cancer immunotherapy. Scientists at Gladstone Institutes have identified two molecules, Sprouty 1 and Sprouty 2, that modify the survival of effector T cells and the development of memory CD8 T cells, which are specialized cells that kill infected cells and play a role in CAR T cancer immunotherapy. A research paper published in PNAS suggests new ways to increase the number, survival, and function of memory CD8 T cells, which could provide better protection against tumors and pathogenic infections. According to the researchers, the findings offer promising potential for immunotherapeutic strategies to combat cancer and chronic infections.

Reprogrammable nanodevices for gene expression control. Researchers at Osaka University have built “gene nanochips” that can switch genes on or off according to the environment, and can be reprogrammed with UV irradiation. The scientists conducted proof-of-concept experiments, described in a study published in Nature Nanotechnology, using artificial cells that produced the diagnostics and reactants (the desired RNA and protein) in a confined nanochip, suggesting the potential of autonomous nanochips in future medical prevention and care.

In-body GPS for high-precision health monitoring and medicine. MIT scientists have developed a system that can pinpoint the location of ingestible implants inside the body with centimeter-level accuracy using low-power wireless signals. The system, dubbed ReMix and promoted as "a GPS for inside the body," is described in a research paper presented at ACM SIGCOMM in Budapest last week. The ability to continuously sense inside the human body could lead to important advances in health monitoring and new therapies. One potential application for ReMix is in proton therapy, a type of cancer treatment that involves bombarding tumors with beams of magnet-controlled protons and requires high precision.