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Genome editing for Duchenne muscular dystrophy: a glimpse of the future?

Circa 2021


As described above, molecular therapeutics enabling expression of a truncated dystrophin have been far developed. However, an unprecedented opportunity to correct the disease-causing mutation has arisen with the advent of Crispr-Cas9 technology (Fig. 1).

Since the generation of a Cas9-transgenic mouse [28], which allowed for pinpoint gene alterations specifically in organs targeted by AAVs encoding for the corresponding guide RNAs (gRNAs), it became clear that the inevitable course of inherited diseases might be altered by Cas9-mediated correction. Although certain limitations were unmasked early on, such as the preference of non-homologous end-joining (NHEJ) over homology-directed repair (HDR) upon enzymatic cleavage of the double stranded DNA by Cas9, or the packaging capacity of AAVs, muscular dystrophies seemed an ideal target for genome editing. DMD mutations inducing Duchenne muscular dystrophy (DMD) seemed particularly well suited, since internal truncations of the protein may lead to a shortened but stable protein with partial functional restitution and a milder disease progression, as seen in the allelic Becker muscular dystrophy (BMD).

The group of E. Olson was first in showing that correction of the loss-of-function mutation on exon 23 in mdx mouse zygotes is possible [29]. Notably, Cas9 combined with a single gRNA was used to inflict a cut in the vicinity of the mutation, accompanied by a single-stranded oligodeoxynucleotide, was efficient in providing HDR in 7 and NHEJ in 4 of the 11 reported corrected mdx mice. Whereas HDR correction of 41% of genomes in the mosaic mice sufficed for a full restoration of dystrophin expression in the muscles examined, a 17% HDR correction level yielded a 47–60% of muscle fibers expressing dystrophin, indicating a selection advantage of the corrected muscle and satellite cells. Moving DMD correction into the postnatal arena, the same group [30] and others [31,32,33] demonstrated feasibility of an AAV-based systemic Cas9 treatment, albeit in different flavors.

Ax-1, Israel and the ‘New Space’ revolution: Q&A with Israel space chief Uri Oron

Israel punches above its weight in space science and exploration, as it does in most other arenas.

For example, the country developed its own line of orbital rockets, the Shavit (“Comet”) series, which has lofted a number of satellites over the years. And in April 2019, Israel’s Beresheet (“In the Beginning”) mission attempted to put a robotic lander down on the moon, something achieved only by the United States, the USSR/Russia and China — and nearly succeeded.

Space Force looking at what it will take to refuel satellites in orbit

WASHINGTON – The U.S. Space Force in 2025 plans to launch to geostationary orbit three small satellites that will attempt to dock with a propellant tanker so they can be refueled in space.

The idea is to “test out pieces of the refueling infrastructure,” Col. Joseph Roth, director of innovation and prototyping at U.S. Space Systems Command, told SpaceNews last week at the Space Symposium in Colorado Springs.

The $50 million experiment, called Tetra-5, is run by the Space Force’s Space Enterprise Consortium. Bids for the project closed earlier this month.

Maxar eager to launch new satellites amid soaring demand for imagery over Ukraine

WASHINGTON – As Maxar Technologies’ satellites continue to collect images of Russia’s invasion of Ukraine, the company is working with customers so it can allocate more capacity to meet U.S. government needs, said Maxar’s CEO Daniel Jablonsky.

With four satellites in orbit, “a lot of times we don’t have a lot of spare capacity,” Jablonsky said in an interview last week at the Space Symposium in Colorado Springs.

“But we made accommodations with some of our other customers to be able to surge capacity for the U.S. and allies,” he said. The company also gets about 200 requests a day for imagery from news media organizations.

These Solar Cells Produce Electricity at Night

Researchers used radiative cooling to generate enough to power LEDs or charge a cell phone.


NASA has agreed to test startup SpinLaunch’s kinetic launcher, a giant circular accelerator that aims to shoot 200 kilogram satellites into space.

The California-based SpinLaunch’s launcher is located at the Spaceport America facility in New Mexico where it will carry out a test flight with NASA later this year, according to the firm.

Satellite pollution is threatening to alter our view of the night sky

The night sky has been a source of information and wonder since the dawn of humankind — and it looks almost the same now as it did then.

But the night sky as we know it is on the precipice of changing dramatically due to the proliferation of satellites just a few hundred miles above Earth.

“For the first time in human history, we’re not going to have access to the night sky in the way that we’ve seen it,” Samantha Lawler, an assistant professor of astronomy at the University of Regina in Canada, said.

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