Faster and More Efficient High-Tech Therapies

Faster and more efficient gene editing technologies, hydrogels able to repair the heart, and functional 3D printed organs for transplants are on the horizon (see below). Scientists are also advancing toward better understanding of important molecular mechanisms for previously hidden cellular events, which could play a role in future cancer therapies.

Meanwhile, scientists at Scripps Research Institute have shed light on poorly understood brain mechanisms that suppress drug and alcohol cravings, uncovering new insights that may help in the development of better addiction medicines. And National University of Singapore have found that regular tea drinkers have better organized brain regions compared to non-tea drinkers.

Faster and more efficient CRISPR delivery system. Scientists at Wake Forest Institute for Regenerative Medicine have fine-tuned their system to deliver a DNA editing tool to alter DNA sequences and modify gene function. The improved 'hit and run' system, described in a research paper published in Nucleic Acids Research, works faster and is more efficient. It permits packaging together in one lentiviral capsid both essential components (Cas9 protein and guide RNA) for CRISPR-mediated gene editing.

New gene therapy could be used to treat stroke. Penn State scientists have developed a new gene therapy able to turn glial cells (abundant support cells in the brain) into neurons, repairing damage that results from stroke and significantly improving motor function in mice. The new therapy, which uses the NeuroD1 gene, is described in a study published in Molecular Therapy. Once further developed, this NeuroD1-based gene therapy could potentially be used to treat stroke.

Injectable hydrogel repairs heart damage and restores cardiac function. Scientists at UC San Diego and biotech spinoff Ventrix have successfully conducted a first-in-human FDA-approved Phase 1 clinical trial of an injectable hydrogel that aims to repair damage and restore cardiac function in heart failure patients who previously suffered a heart attack. The trial, described in a study published in JACC: Basic to Translational Science, is the first to test a hydrogel designed to repair cardiac tissue. It is also the first to test a hydrogel made from the natural scaffolding of cardiac muscle tissue.

Molecular mechanisms choose between cell survival or cell death. Scientists at St. Jude Children's Research Hospital have identified a molecule that plays a pivotal role in determining the fate of cells under stress. A research paper published in Nature indicates that DDX3X, an enzyme that when mutated is involved in a variety of cancers, helps regulate the immune system's first response. Investigators reported evidence that the availability of DDX3X influences how cells interpret and respond to various stressors with measures meant to ensure cell survival or cell death. The findings suggest a possible new approach for treatment of autoinflammatory and other diseases.

Insulin delivery drug could help brain cancer patients. Researchers at University of Georgia have found that a compound molecule used for drug delivery of insulin could be used to treat glioblastoma, an aggressive, usually fatal form of brain cancer. A study published in FASEB Journal shows that Surfen, a compound molecule used to optimize insulin delivery, can stifle the growth of invasive brain tumors and aid in the reduction or refinement of mainstream treatments, particularly radiation and/or chemo.

Differences between responders and non-responders to cancer immunotherapy. Researchers at Tel Aviv University and Sheba Medical Center have identified major differences between responders and non-responders to immunotherapy cancer treatments for patients with metastatic melanoma, more than half of which do not respond to immunotherapy. In the responders, the researchers found higher levels of proteins associated with lipid metabolism, which led to better recognition by the immune system. A study published in Cell also reports that, using genetic engineering, the researchers were able to silence the mechanism responsible for fatty acid metabolism in melanoma tissue and laboratory mice.

3D printing large vascularized human organ building blocks. Scientists at Wyss Institute for Biologically Inspired Engineering at Harvard have developed a new technology, dubbed SWIFT, able to 3D print vascular channel networks directly into living organ building blocks, enabling the creation of larger tissues approximating the size and function of organs, including heart tissue that beats on its own over a seven-day period. SWIFT, described in a study published in Science Advances, could allow 3D printing large, vascularized human organ building blocks.