CRISPR Gene Therapy Shows Potential for Treating Inherited Vision Loss

“This research demonstrates that CRISPR gene therapy for inherited vision loss is worth continued pursuit in research and clinical trials,” said Pierce, director of the Ocular Genomics Institute and Berman-Gund Laboratory for the Study of Retinal Degenerations at Mass Eye and Ear and Harvard Medical School. “While more research is needed to determine who may benefit most, we consider the early results promising. Hearing from several participants about their excitement at finally being able to see the food on their plates is a significant milestone.”

The clinical trial involved 14 participants, including 12 adults (ages 17 to 63) and two children (ages 10 and 14), all born with a form of Leber Congenital Amaurosis (LCA) caused by mutations in the centrosomal protein 290 (CEP290) gene. They received a single injection of a CRISPR/Cas9 genome editing medicine, EDIT-101, in one eye through a specialized surgical procedure. This trial, which marked the first time a CRISPR-based investigational medicine was administered directly inside the body, primarily assessed safety while also evaluating efficacy.

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Study Across 60 Cities in 32 Countries and Six Continents Reveals: New Species Are All Around Us!

Left: Heba Shabaan, a third-year medical student at Weill Cornell Medical College and Dr. Christopher Mason prepare to swab for microbes in the NYC subway system on June 21, 2020. Right: Subway turnstile being swabbed.

By JULIE GRISHAM, WEILL CORNELL

About 12,000 bacteria and viruses collected in a sampling from public transit systems and hospitals around the world from 2015 to 2017 had never before been identified, according to a study by the International MetaSUB Consortium, a global effort at tracking microbes that is led by Weill Cornell Medicine investigators.

For the study, published on May 26, 2021, in the journal Cell, international investigators collected nearly 5,000 samples over a three-year period across 60 cities in 32 countries and six continents. The investigators analyzed the samples using a genomic sequencing technique called shotgun sequencing to detect the presence of various microbes, including bacteria, archaea (single-celled organisms that are distinct from bacteria), and viruses that use DNA as their genetic material. (Other types of viruses that use RNA as their genetic material, such as SARS-CoV-2, the virus that causes COVID-19, would not have been detected with the DNA analysis methods used in this pre-pandemic study.)

This field of research has important implications for detecting outbreaks of both known and unknown infections and for studying the prevalence of antibiotic-resistant microbes in different urban environments.

Continue reading… “Study Across 60 Cities in 32 Countries and Six Continents Reveals: New Species Are All Around Us!”

Scientists are on a path to sequencing 1 million human genomes and use big data to unlock genetic secrets

BY XAVIER BOFILL DE ROS

The first draft of the human genome was published 20 years ago in 2001, took nearly three years and cost between US$500 million and $1 billion. The Human Genome Project has allowed scientists to read, almost end to end, the 3 billion pairs of DNA bases – or “letters” – that biologically define a human being.

That project has allowed a new generation of researchers like me, currently a postdoctoral fellow at the National Cancer Institute, to identify novel targets for cancer treatments, engineer mice with human immune systems and even build a webpage where anyone can navigate the entire human genome with the same ease with which you use Google Maps.

The first complete genome was generated from a handful of anonymous donors to try to produce a reference genome that represented more than just one single individual. But this fell far short of encompassing the wide diversity of human populations in the world. No two people are the same and no two genomes are the same, either. If researchers wanted to understand humanity in all its diversity, it would take sequencing thousands or millions of complete genomes. Now, a project like that is underway.

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Scientists Have Just Successfully Made A Human-Monkey Hybrid

By DOUG NORRIE 

You’ve seen Planet of the Apes right? Did you like how that movie turned out in the end? I’ll give you the cliff notes. The apes won. Well, it appears scientists didn’t get the memo about what happens when we start supercharging primates or just start combining human-related things with monkeys. And sure, I know that monkeys and apes aren’t the same thing, but for our dystopian sci-fi purposes let’s just call it a wash. Because this latest news is pretty groundbreaking. According to a journal article published in The Cellit turns out that scientists have successfully combined human and monkey embryos for the first time. Good luck world, I think we know how this ends. 

This study that landed monkey and human embryos all mixed up together started like all of these things usually do, with someone trying to solve a medical issue. Sure, fine enough. The problem, in this case, had to do with organ transplants and finding a way to better source said organs for procedures. In doing so, scientists began experimentation around cross-breeding these embryos, finding that those of the monkey allowed the process to speed up. Whether this ultimately becomes a good thing in their findings remains to be seen, but for the time being, they have at least proved it to be possible. 

The article was published on April 15th as part of a larger study in which human stem cells were injected into monkey embryos. The results were nearly immediate with researchers showing signs of growth within more than 100 monkeys. They are titling this process, an ominous one called a chimeric embryo, relating to the chimera of Greek Myth. You might know this bad boy as the combination of a bunch of different animals to form one scary super animal. This isn’t an interpretation, this is something actually quoted in the study. Though they stop short of referencing the exact fire-breathing monster the Greeks came up with that’s equal part lion, goat, and serpent. 

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Don’t drop your diet yet, but scientists have discovered how CRISPR can burn fat

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A personalized therapy for metabolic conditions that are linked to obesity could involve removing a small amount of a person’s fat, transforming it into an energy-burning variation using CRISPR gene-editing, and then re-implanting it into the body, according to researchers from the University of Massachusetts Medical School.

In tests involving mice, the implanted human fat cells helped lower sugar concentrations in the blood and decrease fat in the liver. When the mice were put on a high-fat diet, the ones that had been implanted with the human beige fat only gained half as much weight as those that had been implanted with regular human fat.

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Determining if tumor gene testing can select efficacious precision cancer treatment

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NCI-MATCH is a precision medicine cancer trial that seeks to determine whether matching certain drugs or drug combinations in adults whose tumors have specific gene abnormalities will effectively treat their cancer, regardless of their cancer type. Such discoveries could be eligible to move on to larger, more definitive trials. The trial is led by the ECOG-ACRIN Cancer Research Group. Credit: ECOG-ACRIN Cancer Research Group

Five years ago, the ECOG-ACRIN Cancer Research Group (ECOG-ACRIN) and National Cancer Institute (NCI), part of the National Institutes of Health, jointly launched a very different kind of cancer study. NCI-Molecular Analysis for Therapy Choice (NCI-MATCH or EAY131), the largest precision medicine cancer trial to date, sought to match genetic abnormalities driving patients’ tumors with approved or experimental drugs targeting those defects. The type of cancer did not matter. Nearly 6000 cancer patients quickly joined the trial and contributed their tumor specimens for genomic testing. Now, the Journal of Clinical Oncology is publishing an in-depth look into the tumor gene make-up of these patients. It is the largest data set ever compiled on patients with tumors that have progressed on one or more standard treatments, or with rare cancers for which there is no standard treatment. The information contains significant discoveries that tell physicians and patients more about how to use genomic testing to select the best treatments.

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A new CRISPR technique could fix almost all genetic diseases possible

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A new method, called “prime editing,” could, in principle, correct around
89 percent of the mutations that cause inherited human disease.

A less error-prone DNA editing method could correct many more harmful mutations than was previously possible.

Andrew Anzalone was restless. It was late fall of 2017. The year was winding down, and so was his MD/PhD program at Columbia. Trying to figure out what was next in his life, he’d taken to long walks in the leaf-strewn West Village. One night as he paced up Hudson Street, his stomach filled with La Colombe coffee and his mind with Crispr gene editing papers, an idea began to bubble through the caffeine brume inside his brain.

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Humans hava a ‘Salamander-like’ ability to regenerate damaged body parts, study finds

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Axolotls (pictured) have a remarkable ability to regenerate lost body parts.

Salamanders are renowned for their regenerative capabilities, such as growing back entire limbs. We can’t pull off this biological trick, but new research highlights a previously unknown regenerative ability in humans—one held over from our evolutionary past.

Our bodies have retained the capacity to repair injured or overworked cartilage in our joints, says new research published today in Science Advances. Remarkably, the mechanics of this healing process are practically the same as what’s used by amphibians and other animals to regenerate lost limbs, according to the study.

“We call it our ‘inner salamander’ capacity.”

The scientists who identified this previously unknown human capacity are hopeful their findings could lead to powerful new therapies to treat common joint disorders and injuries, including osteoarthritis. More radically, this healing mechanism “might be exploited to enhance joint repair and establish a basis for human limb regeneration,” the authors wrote in the paper.

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Scientists create a device that can mass-produce human embryoids

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These human embryo-like structures (top) were synthesized from human stem cells; they’ve been stained to illustrate different cell types. Images (bottom) of the “embryoids” in the new device that was invented to make them. Yi Zheng/University of Michigan, Ann Arbor

Scientists have invented a device that can quickly produce large numbers of living entities that resemble very primitive human embryos.

Researchers welcomed the development, described Wednesday in the journal Nature, as an important advance for studying the earliest days of human embryonic development. But it also raises questions about where to draw the line in manufacturing “synthetic” human life.

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Gene-hacking mosquitoes to be infertile backfired spectacularly

 

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Best-Laid Plans

On its surface, the plan was simple: gene-hack mosquitoes so their offspring immediately die, mix them with disease-spreading bugs in the wild, and watch the population drop off. Unfortunately, that didn’t quite pan out.

The genetically-altered mosquitoes did mix with the wild population, and for a brief period the number of mosquitoes in Jacobino, Brazil did plummet, according to research published in Nature Scientific Reports last week. But 18 months later the population bounced right back up, New Atlas reports — and even worse, the new genetic hybrids may be even more resilient to future attempts to quell their numbers.

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Why do people love coffee and beer? It’s the buzz, not the taste, study finds

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“People like the way coffee and alcohol make them feel. That’s why they drink it. It’s not the taste,” a Northwestern University researcher said.

Whether you prefer a Café Latte or a diet soda may actually depend on how the drink makes you feel, rather than how it tastes, a new study finds.

This idea contradicts what scientists previously thought: that our taste genes determined why we preferred one drink over the other.

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CRISPR is now being used on humans in the U.S.

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The gene editing trial has two patients so far.

CRISPR therapies are entering the mainstream.

The first U.S. trial of CRISPR in humans has begun, NPR reported Tuesday. Two patients are currently being treated as part of a University of Pennsylvania study. Per NPR, both have difficult-to-treat forms of cancer and both have relapsed after regular treatments. As part of the trial, researchers are taking immune cells from the patients’ own bodies and editing them with CRISPR before putting them back in. The hope is that these edited cells will be better at identifying and attacking the cancer than their unaltered counterparts. According to the U.S. government clinical trial registry, the researchers are hoping to enroll 18 people in their study. But it’s not certain yet whether they’ll be approved for that many subjects, reports Jon Fingas for Engadget.

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