Lifeboat Foundation

In some mammals, the timing of the normally continuous embryonic development can be altered to improve the chances of survival for both the embryo and the mother. This mechanism to temporarily slow development, called embryonic diapause, often happens at the blastocyst stage, just before the embryo implants in the uterus.

During diapause, the embryo remains free-floating and pregnancy is extended. This dormant state can be maintained for weeks or months before development is resumed, when conditions are favorable. Although not all mammals use this reproductive strategy, the ability to pause development can be triggered experimentally. Whether human cells can respond to diapause triggers remained an open question.

Now, a study by the labs of Aydan Bulut-Karslıoğlu at the Max Planck Institute for Molecular Genetics in Berlin and Nicolas Rivron at the Institute of Molecular Biotechnology (IMBA) of the Austrian Academy of Sciences in Vienna, has identified that the molecular mechanisms that control embryonic diapause also seem to be actionable in human cells.

“We are excited to explore the other trees by applying similar technologies.” first appeared on The Cool Down.

NEW YORK — In a groundbreaking development, scientists have created a revolutionary method to track the spread of cancer throughout the body, potentially paving the way for more effective treatments against this devastating disease. The new technology, developed by researchers at Cold Spring Harbor Laboratory and Weill Cornell Medicine in New York, uses genetic “barcodes” to monitor the movement of individual cancer cells, providing unprecedented insights into the process of metastasis.

In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the development of the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) systems. In 2020, the Nobel Prize in Chemistry was awarded to the scientists for the discovery of CRISPR–Cas9 system, a revolutionary genome editing technology that advanced DNA therapeutics. Subsequently, the CRISPR–Cas13 system has emerged as a potential tool to identify and rectify errors in RNA sequences. CRISPR–Cas13 is a novel technology is specifically engineered for virus detection and RNA-targeted therapeutics. The CRISPR RNA (CrRNA) targets specific and non-specific RNA sequences, and Cas13 is an effector protein that undergoes conformational changes and cleaves the target RNA. This RNA-targeting system holds tremendous promise for therapeutics and presents a revolutionary tool in the landscape of molecular biology.

Now, in a recently published BioDesign Research study, a team of researchers led by Professor Yuan Yao from ZJU-Hangzhou Global Scientific and Technological Innovation Center, Zhejiang University, China has elucidated the latest research trends of CRISPR–Cas13 in RNA-targeted therapies. Talking about this paper, which was published online on 6 September 2024, in Volume 6 of the journal, Prof. Yao says, “By focusing on RNA-;the intermediary between DNA and proteins-;CRISPR-Cas13 allows scientists to temporarily manipulate gene expression without inducing permanent changes to the genome. This flexibility makes it a safer option in scenarios where genome stability is critical.”

RNA plays a central role in carrying genetic information from DNA to protein-synthesizing machinery, and also regulates gene expression and participates in numerous cellular processes. Defects in RNA splicing or mutations can lead to a wide variety of diseases, ranging from metabolic disorders to cancer. A point mutation occurs when a single nucleotide is erroneously inserted, deleted, or changed. CRISPR–Cas13 plays a role in identifying and correcting these mutations by employing REPAIR (RNA editing for programmable A-to-I replacement) and RESCUE (RNA editing for specific C-to-U exchange) mechanisms. Explaining the applications of Cas13-based gene editors, Prof. Yao adds, “The mxABE editor, for example, can be used to correct a nonsense mutation linked with Duchenne muscular dystrophy that can be corrected with mxABE. This approach has proved high editing efficiency, restoring dystrophin expression to levels more than 50% of those of the wild type.”

Imagine being one cartwheel away from changing your appearance. One flip, and your brunette locks are platinum blond. That’s not too far from what happens in some prokaryotes, or single-cell organisms, such as bacteria, that undergo something called inversions.

A study led by scientists at Stanford Medicine has shown that inversions, which cause a physical flip of a segment of DNA and change an organism’s genetic identity, can occur within a single gene, challenging a central dogma of biology — that one gene can code for only one protein.

“Bacteria are even cooler than I originally thought, and I’m a microbiologist, so I already thought they were pretty cool,” said Rachael Chanin, PhD, a postdoctoral scholar in hematology. Microbiologists have known for decades that bacteria can flip small sections of their DNA to activate or deactivate genes, Chanin said. To the team’s knowledge, however, those somersaulting pieces have never been found within the confines of a single gene.

Robert C Williams performed genetic analysis to understand how the HLA and SLC16A11 genes affect Type 2 diabetes in Indigenous Americans.

GC Therapeutics’ plug-and-play stem cell programming platform aims to reduce cell therapy development time by up to 100 times.

Cell therapies have revolutionized the treatment of certain disease areas; however, challenges in scaling these therapies…

Cell therapy startup GC Therapeutics (GCTx) has emerged from the lab of renowned geneticist George Church, securing a $65 million Series A funding round that brings the total raised by the company to a cool $75 million. The company is on a mission to enable the next generation of cell therapies through its proprietary TFome platform, which GCTx claims is the first plug-and-play induced pluripotent stem cell (iPSC) cellular programming platform.

Continue reading “George Church lab spawns $75m cell therapy startup” »

RNA interference (RNAi) technology has gradually become a cutting-edge technology for treating diseases such as genetic disorders and cancer due to its huge potential in gene expression regulation. However, the efficient delivery and safety of short interfering RNA (siRNA) remain key challenges for its clinical application.

In the next couple of decades, we will be able to do things that would have seemed like magic to our grandparents.

This phenomenon is not new, but it will be newly accelerated. People have become dramatically more capable over time; we can already accomplish things now that our predecessors would have believed to be impossible.

We are more capable not because of genetic change, but because we benefit from the infrastructure of society being way smarter and more capable than any one of us; in an important sense, society itself is a form of advanced intelligence. Our grandparents – and the generations that came before them – built and achieved great things. They contributed to the scaffolding of human progress that we all benefit from. AI will give people tools to solve hard problems and help us add new struts to that scaffolding that we couldn’t have figured out on our own. The story of progress will continue, and our children will be able to do things we can’t.