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Category: biotech/medical – Page 316

Through years of engineering gene-editing systems, researchers have developed a suite of tools that enable the modification of genomes in living cells, akin to “genome surgery.” These tools, including ones based on a natural system known as CRISPR/Cas9, offer enormous potential for addressing unmet clinical needs, underscored by the recent FDA approval of the first CRISPR/Cas9-based therapy.

A relatively new approach called “prime editing” enables gene-editing with exceptional accuracy and high versatility, but has a critical tradeoff: variable and often low efficiency of edit installation. In other words, while prime edits can be made with high precision and few unwanted byproducts, the approach also often fails to make those edits at reasonable frequencies.

In a paper that appeared in print in the journal Nature on April 18, 2024, Princeton scientists Jun Yan and Britt Adamson, along with several colleagues, describe a more efficient prime editor.

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Instead of creating materials that are made to last, Freeman says their materials are made to task — perform a specific function and then modify themselves to serve a new function.

This achievement holds significant promise for advancements in regenerative medicine, drug delivery methods, and diagnostic technologies.

“With this discovery, we can think of engineering fabrics or tissues that can be sensitive to changes in their environment and behave in dynamic ways,” states Freeman.

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Recent advancements in our comprehension of human health and disease have been propelled by pioneering research utilizing in vitro 3D cell culture models, including both single-cell spheroids and multicellular organoids.

The refinement of these 3D cell culture models hinges on the capacity to visualize, measure, and track their development and expansion over time. Nonetheless, the methods employed to evaluate and scrutinize these intricate cell models are not without their challenges.

This video explores the challenges associated with characterizing organoids and introduces some solutions to these challenges.

Learn more about our solutions: https://www.sartorius.com/en/applicat…

Continue reading “Challenges in Producing and Analyzing Organoids” | >

Scientists at the Leibniz Institute of Plant Biochemistry (IPB) have succeeded for the first time in stably and precisely inserting large gene segments into the DNA of higher plants very efficiently. To do this, they optimized the gene-editing method CRISPR/Cas, commonly known as “genetic scissors.”

The improved CRISPR method offers great opportunities for the targeted modification of genes in higher plants, both for breeding and research. The study, led by Prof. Alain Tissier and Dr. Tom Schreiber, has been published in Molecular Plant.

CRISPR/Cas is a method with enormous potential for the targeted modification of individual genes. However, this does not apply to all kinds of genetic modifications that breeders and scientists have on their wish lists. While the genetic scissors are ideal for knocking out genes, i.e., switching off or removing existing genes, they do not work well for precisely inserting genes or replacing gene segments. To date, genetic scissors have been too inefficient and therefore of little use for the targeted insertion of genes into the DNA of higher plants.

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A clinical trial led by Weill Cornell Medicine investigators showed that a nasal spray that patients administer at home, without a physician, successfully and safely treated recurrent episodes of a condition that causes rapid abnormal heart rhythms. The study, published March 25 in the Journal of the American College of Cardiology, provides real-world evidence that a wide range of patients can safely and effectively use the experimental drug, called etripamil, to treat recurrent paroxysmal supraventricular tachycardia (PSVT) episodes at home, potentially sparing them the need for repeated hospital trips for more invasive treatments.

The study is the latest in a series of studies by lead author Dr. James Ip, professor of clinical medicine at Weill Cornell Medicine and a cardiologist at New York-Presbyterian/Weill Cornell Medical Center, and colleagues to demonstrate the potential of nasal spray calcium-channel blocker etripamil as an at-home treatment PSVT.

Dr. Ip received compensation as a steering committee member for Milestone Pharmaceuticals, the maker of etripamil and sponsor of the trial.

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In a new study, a Yale Pathology team has identified a possible therapeutic target for treating obesity-induced liver cancer.

Researchers say inhibiting a molecule called fatty acid binding protein 5 (FABP5) could block tumor progression in many cases:

Inhibiting a certain protein in mice reduced obesity-induced liver tumor development, Yale researchers found. It could reveal a future treatment route.

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