Mount Sinai Launches Large-Scale Genetic Sequencing Project with the Regeneron Genetics Center; Massive Project Aims to Enroll One Million Racially and Ethnically Diverse Patients, Advance Precision Medicine Research, and Improve Patient Care

On August 12, 2022, it was announced that Mount Sinai Health System and the Icahn School of Medicine at Mount Sinai have launched a new human genome sequencing research project called the Mount Sinai Million Health Discoveries Program with the Regeneron Genetics Center (RGC), part of the industry-leading, New York-based biotechnology company Regeneron. The Program aims to enroll one million Mount Sinai patients over a five-year period, making it one of the most ambitious projects of its kind and the largest Regeneron-supported sequencing effort to date. Its goal is to provide researchers with a unique data set that will help them assess the true potential of genetics-based, precision medicine approaches to guide everyday patient care, as well as to generate new insights to guide the discovery and development of potential new therapies. The collaboration team plans to combine the RGC’s massive gene sequencing capabilities and scientific research expertise with Mount Sinai’s large, diverse patient population and advanced electronic health records systems, all supported by a digital health platform developed by Vibrent Health.

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Columbia-Led Research Key to FDA’s Recent Approval of First Systemic Treatment (Olumiant) for Hair Loss in Alopecia Areata

(L-R) Dr. Julian Mackay-Wiggan, Dr. Raphael Clynes, Dr. Angela Christiano. (Credit: Columbia University Irving Medical Center)

For over a decade, Columbia University geneticist Angela Christiano, PhD, has attended the annual meeting of the National Alopecia Areata Foundation, where hundreds of individuals affected by the hair loss disorder gather to support one another and learn about the latest scientific research. The meeting is a safe space where patients with alopecia, many of whom have lost all their hair, joyfully remove their wigs and head coverings for the three-day celebration, without fear of shame or judgment. But this year’s meeting was a bit different. Dr. Christiano had trouble recognizing conference attendees she’s known and worked with for years, because many of them now have full heads of hair. For people with alopecia areata, an autoimmune disease that can cause hair loss so complete that people even lose their eyebrows, the change in appearance was dramatic.

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Stiff, Achy Knees? New Lab-Made Cartilage Gel Outperforms Real Thing

Over-the-counter pain relievers, physical therapy, steroid injections–some people have tried it all and are still dealing with knee pain. Often knee pain comes from the progressive wear and tear of cartilage known as osteoarthritis, which affects nearly one in six adults–867 million people–worldwide. For those who want to avoid replacing the entire knee joint, there may soon be another option that could help patients get back on their feet fast, pain-free, and stay that way. Writing in the journal Advanced Functional Materials, a Duke University-led team says its members have created the first gel-based cartilage substitute that is even stronger and more durable than the real thing. The article, published on August 4, 2022, is titled” A Synthetic Hydrogel Composite with a Strength and Wear Resistance Greater Than Cartilage.”

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Positive Results of Preclinical Study Demonstrate Tolerizing Vaccine Efficacy in Relapsing-Remitting Model of Multiple Sclerosis

On August 11, 2022, Pasithea Therapeutics Corp. (Nasdaq: KTTA), a biotechnology company focused on the discovery, research, and development of new and effective treatments for psychiatric and neurological disorders, announced positive results from a preclinical proof of concept study of PAS002, its tolerizing vaccine program in multiple sclerosis (MS). Earlier this year, a study in Nature, the world’s leading science journal, showed that a molecule called GlialCAM found in the brain’s white matter is attacked in MS. GlialCAM shares a component of its protein structure that mimics an identical component of the Epstein Barr Virus (“EBV”) Nuclear Antigen-1, which plays a critical role in triggering MS.

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Panacell Biotech to Conduct Toxicity Tests for NK Cells, Exosomes, and Brown Adipose-Derived Stem Cells to Treat Patients with Long COVID

On August 10, 2022, Panacell Biotech Co., Ltd. said that natural killer (NK) cells, exosomes, and brown adipose-derived stem cells are effective to treat patients with long COVID conditions, or post COVID-19 conditions, as well as those with terminal illness. Panacell Biotech is South Korea’s research institute specializing in advanced regenerative medical cell therapy using adipose-derived stem cells (ADSC). The company announced on August 10,2022 that it will soon conduct those cells’ and exosomes’ toxicity tests through clinical trials and laboratory animals.

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Scientists Identify Novel Molecular Biomarkers in Cells That Spread Triple-Negative Breast Cancer; May Serve As Therapeutic Targets

Studying a deadly type of breast cancer called triple-negative, Johns Hopkins Medicine scientists say they have identified key molecular differences between cancer cells that cling to an initial tumor and those that venture off to form distant tumors. The research, using mouse models and human tissues, could pave the way for developing new treatments that target such molecular variations. A report on the findings was published August 3, 2022 in Science Translational Medicine. The article is titled Triple-Negative Breast Cancer Metastasis Involves Complex Epithelial-Mesenchymal Transition Dynamics and Requires Vimentin.” The subject served as the cover story of the August 3, 2022 online issue of Science Translational Medicine.

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A Brain Mechanism Underlying Evolution of Anxiety; Creating Mouse Models with Human-Type Mutations in Vesicular Monoamine Transporter 1 (VMAT1) Enables Assessment of Their Roles in Genetic, Neuronal, and Behavioral Mechanisms

Monoamine neurotransmitters such as serotonin and dopamine play important roles in our cognitive and emotional functions. Their evolutionary origins date back to metazoans, and while the function of related genes is strongly evolutionarily conserved, genetic variation within and between species has been reported to have a significant impact on animal mental characteristics such as sociality, aggression, anxiety, and depression.

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E. coli Engineered from Stool Samples Can Survive Hostile Gut Environment Long Enough to Treat Disease

Scientists have long tried to introduce genetically engineered bacteria into the gut to treat diseases. In the past, these attempts have focused on engineering common lab strains of E. coli, which cannot compete with the native gut bacteria that are well adapted to their host. Now, a group of researchers from the University of California, San Diego, successfully engineered E. coli collected from both human and mice gut microbiomes and showed that they have the potential to treat diseases such as diabetes. Their finds are publishing in Cell on August 4, 2022. The open-access article is titled “Intestinal Transgene Delivery with Native E. coli Chassis Allows Persistent Physiological Changes.”

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A New Step Towards Using Magnetic Bacteria in Cancer Treatment

Imagine a tiny vehicle, a nanocar (one million times smaller than a millimeter), equipped with a magnetic structure that allows it to be controlled and steered by means of magnetic fields. Let’s imagine that we can insert this car into the human body and send it to the exact spot where a drug needs to be released or cancer cells need to be eliminated. Numerous scientists across the world are working on this bold idea, including the multidisciplinary Magnetism and Magnetic Materials group (GMMMT) at the UPV/EHU-University of the Basque Country. This team is involved in research that was published on April 26, 2022 in the journal ACS Nano and is taking a new step towards turning the idea into reality. The article is titled “Magnetic Anisotropy of Individual Nanomagnets Embedded in Biological Systems Determined by Axi-Asymmetric X-ray Transmission Microscopy.

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New DNA Repair Kit Using Baculovirus Delivery System Successfully Corrects Hereditary Kidney Disease Defect in Patient-Derived Cells

Genetic mutations which cause a debilitating hereditary kidney disease affecting children and young adults have been fixed in patient-derived kidney cells using a potentially game-changing DNA repair-kit. The advance, developed by University of Bristol (UK) scientists, was published on July 22, 2022 in Nucleic Acids Research. The open-access article is titledHighly Efficient CRISPR-Mediated Large DNA Docking and Multiplexed Prime Editing Using a Single Baculovirus.” In this new study, the international team describe how they created a DNA repair vehicle to genetically fix faulty podocin, a common genetic cause of inheritable steroid-resistant nephrotic syndrome (SRNS).

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