Scientists use gene therapy and a novel light-sensing protein to restore vision in mice

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy. The National Eye Institute, part of the National Institutes of Health, provided a Small Business Innovation Research grant to Nanoscope, LLC for development of MCO1. The company is planning a U.S. clinical trial for later this year.

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AAOS Advances Biologics Initiative with Innovative Dashboard

The American Academy of Orthopaedic Surgeons (AAOS) continues to demonstrate its commitment to advancing the quality of musculoskeletal care in a fully transparent and scientific way. Debuting today as a new member benefit, the AAOS Biologics Dashboard is a dynamic online tool designed to help orthopaedic surgeons navigate the approval status of biologic-based interventions. The development of the AAOS Biologics Dashboard is just one of several efforts within the Academy’s Biologics Initiative that offers evidence-based guidance to the musculoskeletal health community. An additional effort is the revision of two biologics-related position statements, recently approved by the AAOS Board of Directors.

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Exosome treatment improves recovery from heart attacks in a preclinical study

Research in pigs shows that using the exosomes naturally produced from a mix of heart muscle, endothelial and smooth muscle cells — all derived from human induced pluripotent stem cells — yields regenerative benefits equivalent to the injected human induced pluripotent stem cell-cardiac cells.

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Could a tiny fish hold the key to curing blindness?

Imagine this: A patient learns that they are losing their sight because an eye disease has damaged crucial cells in their retina. Then, under the care of their doctor, they simply grow some new retinal cells, restoring their vision.

Although science hasn’t yet delivered this happy ending, researchers are working on it – with help from the humble zebrafish. When a zebrafish loses its retinal cells, it grows new ones. This observation has encouraged scientists to try hacking the zebrafish’s innate regenerative capacity to learn how to treat human disease. That is why among the National Eye Institute’s 1,200 active research projects, nearly 80 incorporate zebrafish.

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Scientists uncover a novel approach to treating Duchenne muscular dystrophy

Scientists at Sanford Burnham Prebys, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition.

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Scientists show MRI predicts the efficacy of a stem cell therapy for brain injury

Scientists at Sanford Burnham Prebys Medical Discovery Institute and Loma Linda University Health have demonstrated the promise of applying magnetic resonance imaging (MRI) to predict the efficacy of using human neural stem cells to treat a brain injury—a first-ever “biomarker” for regenerative medicine that could help personalize stem cell treatments for neurological disorders and improve efficacy. The study was published in Cell Reports.

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