A new gene editor takes advantage of CRISPR-associated proteins to insert whole genes into the genome, scientists report.
Category: genetics – Page 51
Advanced gene editor enables more precise insertion of complete genes
Ask scientists which gene-editing tool is most needed to advance gene therapy, and they’d probably describe a system that’s now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.
The gene editor—called evoCAST—goes a long way toward solving a problem that has confounded the development of gene therapies from the field’s beginnings: How to add long stretches of DNA to defined locations in the human genome without creating unwanted modifications.
The latest iteration of the editor, which utilizes complex enzymes found in bacteria, can be programmed to insert an entire gene—or multiple genes—into a specific location in the human genome with an efficiency suitable for gene therapy. Details of the editor are described in a paper published in Science.
Abstract: For several years, researchers have known that hyperactivity of a subset of neurons located in the hypothalamus, called AgRP neurons, is common in mice with diabetes
“These neurons are playing an outsized role in hyperglycemia and type 2 diabetes,” said UW Medicine endocrinologist Dr. Michael Schwartz, corresponding author of the paper.
To determine if these neurons contribute to elevated blood sugar in diabetic mice, researchers employed a widely used viral genetics approach to make AgRP neurons express tetanus toxin, which prevents the neurons from communicating with other neurons.
Unexpectedly, this intervention normalized high blood sugar for months, despite having no effect on body weight or food consumption.
Conventional wisdom is that diabetes, particularly type 2 diabetes, stems from a combination of genetic predisposition and lifestyle factors, including obesity, lack of physical activity and poor diet. This mix of factors leads to insulin resistance or insufficient insulin production.
Until now, scientists have traditionally thought the brain doesn’t play a role in type 2 diabetes, according to Schwartz.
The paper challenges this and is a “departure from the conventional wisdom of what causes diabetes,” he said.
The new findings align with studies published by the same scientists showing that injection of a peptide called FGF1 directly into the brain also causes diabetes remission in mice. This effect was subsequently shown to involve sustained inhibition of AgRP neurons.
Q&A: A generative AI technique for designing RNA with improved function
Ribonucleic acid, also called RNA, is a molecule present in all living cells. It plays a critical role in transmitting genetic instructions from DNA and creating proteins. With the power to execute a plethora of functions, the little RNA “messenger” has led to important innovations across therapeutics, diagnostics, and vaccines, and made us rethink our understanding of life itself.
A team of researchers from Boston University’s Biological Design Center and the Department of Biomedical Engineering recently made significant steps forward in the development of the next generation of computational RNA tools. They recently published a study in Nature Communications describing a generative AI technique for designing different types of RNA molecules with improved function.
Much like a large language model that can be used to compose entirely new texts, the model can compose new RNA sequences tailored for specific tasks in the cell or in a diagnostic assay. Their research has shown that it’s possible to predict and generate RNA sequences that have specific functions across a broad array of potential applications.
World’s First CRISPR-Edited Spider Produces Glowing Red Silk From Its Spinneret
One of the reasons why this has never happened before is that spiders themselves are difficult organisms to work with within the laboratory. They are a diverse group, have a complex genome structure, and their cannibalistic nature means that they have to be reared individually, otherwise their cage neighbors would be gobbled up. Despite this, new developments in Parasteatoda tepidariorum have allowed this species to become a research model.
The research team looked into spider silk as the target. Spider silk is an incredibly strong and scientifically interesting substance, as it is five times stronger than a steel cable of the same weight, tear-resistant, while also being biodegradable, lightweight, and elastic.
To genetically modify this arachnophobe’s nightmare, the scientists developed an injection solution. This had a gene-editing system that also included a red fluorescent protein gene sequence. This solution was then injected into oocytes inside unfertilized female spiders, when these spiders mated with males, it resulted in the genetically modified offspring.
Which Epigenetic Clock Is Best For Evaluating Death Risk?
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From sequence to structure: A fast track for RNA modeling
In Biology 101, we learn that RNA is a single, ribbon-like strand of base pairs that is copied from our DNA and then read like a recipe to build a protein. But there’s more to the story. Some RNA strands fold into complex shapes that allow them to drive cellular processes like gene regulation and protein synthesis, or catalyze biochemical reactions.
We know that these active molecules, called non-coding RNAs, are present in all life forms, yet we’re just starting to understand their many roles—and how they can be harnessed for applications in environmental science, agriculture, and medicine.
To study—and potentially modify—the functions of non-coding RNAs, we need to determine their structure. Scientists from Lawrence Berkeley National Laboratory (Berkeley Lab) and the Hebrew University of Jerusalem have developed a streamlined process that predicts the structure of an RNA molecule down to the atomic level.
