Lifeboat Foundation

:33333 could lead to future cures of muscular dystrophy.

Facioscapulohumeral muscular dystrophy (FSHD) is caused by altered expression of DUX4, a gene important during development that is not usually present in adult cells. In FSHD skeletal muscle, activation of DUX4 leads to apoptosis. To identify potential targets that mediate DUX4-induced cell death, Lek et al. performed an unbiased screen using CRISPR-Cas9. Hypoxia signaling emerged as a target, and treating patient cells and zebrafish models of FSHD with inhibitors of hypoxia signaling reduced cell death and expression of DUX4 target genes and improved structural defects and muscle function. Results demonstrate the utility of this CRISPR-Cas9 screen for identifying putative therapeutic targets for FSHD.

The emergence of CRISPR-Cas9 gene-editing technologies and genome-wide CRISPR-Cas9 libraries enables efficient unbiased genetic screening that can accelerate the process of therapeutic discovery for genetic disorders. Here, we demonstrate the utility of a genome-wide CRISPR-Cas9 loss-of-function library to identify therapeutic targets for facioscapulohumeral muscular dystrophy (FSHD), a genetically complex type of muscular dystrophy for which there is currently no treatment. In FSHD, both genetic and epigenetic changes lead to misexpression of DUX4, the FSHD causal gene that encodes the highly cytotoxic DUX4 protein. We performed a genome-wide CRISPR-Cas9 screen to identify genes whose loss-of-function conferred survival when DUX4 was expressed in muscle cells. Genes emerging from our screen illuminated a pathogenic link to the cellular hypoxia response, which was revealed to be the main driver of DUX4-induced cell death.

Scientists took conspiracy theories seriously and analyzed the coronavirus to reveal its natural origins.

:ooooo.

Unlocking the full potential of cannabis for agriculture and human health will require a co-ordinated scientific effort to assemble and map the cannabis genome, says a just-published international study led by University of Saskatchewan researchers.

In a major statistical analysis of existing data and studies published in the Annual Review of Plant Biology, the authors conclude there are large gaps in the scientific knowledge of this high-demand, multi-purpose crop.

Continue reading “Mapping the cannabis genome to improve crops and health” »

Researchers have developed the first computational model of a human cell and simulated its behavior for 15 minutes—the longest time achieved for a biological system of this complexity. In a new study, simulations reveal the effects of spatial organization within cells on some of the genetic processes that control the regulation and development of human traits and some human diseases.

The study, which produced a new computational platform that is available to any researcher, is published in the journal PLOS Computational Biology.

“This is the first program that allows researchers to set up a virtual human cell and change chemical reactions and geometries to observe cellular processes in real time,” said Zhaleh Ghaemi, a research scientist at the University of Illinois at Urbana-Champaign and lead author of the study.

The Curious Case of Sickle-Cell Anemia

Even those uninterested in biology have likely heard of the disorder. Sickle-cell anemia holds the crown as the first genetic disorder to be traced to its molecular roots nearly a hundred years ago.

The root of the disorder is a single genetic mutation that drastically changes the structure of the oxygen-carrying protein, beta-globin, in red blood cells. The result is that the cells, rather than forming their usual slick disc-shape, turn into jagged, sickle-shaped daggers that damage blood vessels or block them altogether. The symptoms aren’t always uniform; rather, they come in “crisis episodes” during which the pain becomes nearly intolerable.

The longfin inshore squid can edit the RNA inside its nerve cells, Wired reports, meaning that it can drastically alter the behavior of its biological machinery as needed — perhaps to help the animal rapidly adapt to new environments. It’s a bizarre discovery, and one that could potentially lead to better genetic treatments for humans.

Neat Trick

Researchers from the Marine Biological Laboratory found that the squid alters the RNA within its axons instead of the DNA within its nuclei, according to research published Monday in the journal Nucleic Acids Research. Thus far, it’s the only animal known to do so.

Researchers at Stanford University have demonstrated they can rejuvenate human cells, making them more like young cells again, by rewinding an epigenetic aging clock.

Using new gene-editing technology, researchers have rewired mouse stem cells to fight inflammation caused by arthritis and other chronic conditions. Such stem cells, known as SMART cells (Stem cells Modified for Autonomous Regenerative Therapy), develop into cartilage cells that produce a biologic anti-inflammatory drug that, ideally, will replace arthritic cartilage and simultaneously protect joints and other tissues from damage that occurs with chronic inflammation.

Coronavirus RNA survived for up to 17 days aboard the Diamond Princess cruise ship, living far longer on surfaces than previous research has shown, according to new data published Monday by the Centers for Disease Control and Prevention.

The study examined the Japanese and U.S. government efforts to contain the COVID-19 outbreaks on the Carnival-owned Diamond Princess ship in Japan and the Grand Princess ship in California. Passengers and crew on both ships were quarantined on board after previous guests, who didn’t have any symptoms while aboard each of the ships, tested positive for COVID-19 after landing ashore.

The RNA, the genetic material of the virus that causes COVID-19, “was identified on a variety of surfaces in cabins of both symptomatic and asymptomatic infected passengers up to 17 days after cabins were vacated on the Diamond Princess but before disinfection procedures had been conducted,” the researchers wrote, adding that the finding doesn’t necessarily mean the virus spread by surface.