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Category: biotech/medical – Page 1,481

In the latest of ongoing efforts to expand technologies for modifying genes and their expression, researchers have developed chemically modified guide RNAs for a CRISPR system that targets RNA instead of DNA. These chemically-modified guide RNAs significantly enhance the ability to target — trace, edit, and/or knockdown — RNA in human cells.

In a study published today in Cell Chemical Biology, the team explores a range of different RNA modifications and details how the modified guides increase efficiencies of CRISPR activity from 2-to 5-fold over unmodified guides. They also show that the optimized chemical modifications extend CRISPR targeting activity from 48 hours to four days. The researchers worked in collaboration with scientists at Synthego Corporation and New England BioLabs, bringing together a diverse team with expertise in enzyme purification and RNA chemistry. To apply these optimized chemical modifications, the research team targeted cell surface receptors in human T cells from healthy donors and a “universal” segment of the genetic sequence shared by all known variants of the RNA virus SARS-COV-2, which is responsible for the COVID-19 pandemic.

Increasing the efficiencies and “life” of CRISPR-Cas13 guides is of critical value to researchers and drug developers, allowing for better gene knockdown and more time to study how the gene influences other genes in related pathways.

“CRISPR RNA guide delivery can be challenging, with knockdown time limited due to rapid guide degradation. We were inspired by the guide modifications developed for other DNA-targeting CRISPRs and wanted to test if chemically modified guides could improve knockdown time for RNA-targeting CRISPR-Cas13 in human cells,” says Alejandro Méndez-Mancilla, PhD, a postdoctoral scientist in the lab and co-first author of the study.

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“The reason a glucose-responsive insulin is important is that the biggest barrier to the effective use of insulin, especially in Type 1 diabetes, is the fear of the consequences of blood sugar going too low,” says study author Michael A. Weiss.

For sufferers of diabetes, keeping blood glucose levels within a healthy range can be a difficult and labor-intensive balancing act that often requires regular insulin injections, but some scientists imagine a future where medicine does the heavy lifting for them. A team at Indiana University School of Medicine has taken a promising step towards this future, demonstrating a type of “synthetic hinge” that swings into action when blood glucose levels call for corrective action.

The hormone insulin plays a vital role in keeping glucose at healthy levels in the blood, pulling it out of the bloodstream and helping turn it into energy. In diabetes patients, insufficient amounts or insulin that results in a reduction in effectiveness means that blood glucose levels are left to rise to potentially dangerous levels, which can have serious consequences.

Injections of insulin are a way for Type 1 diabetics to manage the condition, but one dangerous side effect of this is the potential for them to drive blood-sugar levels too low, a condition known as hypoglycemia. These concerns have moved scientists to explore a concept known as “glucose-responsive insulin,” an engineered form of the hormone that would self-adjust depending on the blood sugar levels of the patient.

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The idea is to offer the predictions for the structure of practically every protein with a known sequence of amino acids free of charge. “We believe that this is the most important contribution to date that artificial intelligence has contributed to scientific knowledge,” he said following the publication of DeepMind’s research in the medical journal Nature.

DeepMind, a company bought by Google, predicts with unprecedented precision the 3D structure of nearly all the proteins made by the human body.

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The company that makes SG was testing it mainly against other cancers, but Dr. Tagawa, who is also a professor of urology and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine, and his colleagues showed in a 2015 pilot trial that it shrank tumors in three of six patients with advanced urothelial carcinoma that had not responded to platinum-based chemotherapy. That trial led to the enrollment of a group of 45 patients with treatment-refractory urothelial carcinoma (amongst many others with various advanced cancers) with encouraging results.

A new treatment for advanced urothelial cancer was effective with tolerable side effects in an international, multi-center phase 2 clinical trial led by investigators at Weill Cornell Medicine and NewYork-Presbyterian.

The trial results prompted a U.S. Food and Drug Administration (FDA) accelerated approval of the treatment on April 13, giving patients with this very aggressive type of cancer a new therapeutic option.

In the study, published online April 30 in the Journal of Clinical Oncology, the researchers gave the treatment, sacituzumab govitecan (SG), previously known as IMMU-132 and now by the trade name Trodelvy, to 113 patients with advanced urothelial carcinoma, the most common type of bladder cancer. The trial population had progressed despite treatment with platinum-based chemotherapy and immune-boosting checkpoint inhibitors, and overall had a median of 3 prior lines of therapy. Treatment with SG was followed by sustained reductions in tumor size for 31 patients (27 percent), including complete tumor disappearance in six patients. The most common severe side effects included very low white blood cell count in 34 percent (with fever in 10 percent) and severe diarrhea in 9 percent, which were managed with dose adjustment and best supportive care.

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Right now, protection against Covid-19 comes via an injection. But in future, those vaccines could come from inhalers or even pills.

In a white, airy laboratory in Medicon Village, one of southern Sweden’s largest science parks, chemist Ingemo Andersson holds up a thin, plastic inhaler, half the size of a matchbox.

Her team is hoping this tiny product could play a big role in the global fight against coronavirus allowing people to take powdered versions of future vaccines at home.

Researchers are looking at easier ways for people to get the vaccine, including via inhalers and tablets.

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The news we like: “In five to 10 years time from now, we’ll have a new, special kind of drugs: longevity drugs. And unlike today’s medication, which always focused on one disease, this kind of drug will will give us an opportunity to influence aging as a whole and a very fatalistic way, working on healthspan, not only on lifespan… it’s very likely that this new drug will be developed with the help of artificial intelligence, which will compress drug development cycle by two or three times from what they are today.”

Ahead of the launch of his new book Growing Young, Sergey Young joins us for a video interview to discuss longevity horizons, personal health strategies and disruptive tech – and how we are moving towards radically extending our lifespan and healthspan.

Sergey Young, the longevity investor and founder of the Longevity Vision Fund is on a mission to extend healthy lifespans of at least one billion people. His new book, Growing Young, is released on 24th August and is already rising up the Amazon charts.

“It’s been amazing three years journey,” Young told Longevity. Technology. “I spent hours and days in different labs in the best clinics in the world and best academic institutions. I even talked to Peter Jackson! I’m very excited to share with everyone, so every reader can start their longevity journey today.”

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