CRISPR & Genetic Engineering News and Discussions

weatheriscool
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Yuli Ban wrote: Sun Jun 13, 2021 3:56 am


Genetic engineering is probably what allows for life extension, the end of cancer, heart disease and hell maybe a vastly superior human species if we're smart. It is very promising.
weatheriscool
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New drugs may kill and limit reproduction of bowel cancer cells

by Hudson Institute of Medical Research
https://medicalxpress.com/news/2021-06- ... ancer.html
Drugs that are being trialed to treat leukemia could also be used to fight bowel cancer after a breakthrough by Hudson Institute of Medical Research scientists.

In a world-first, researchers found that the drugs could potentially be used to fight bowel cancer, using Nobel Prize-winning genetic screening technology CRISPR.

The researchers were using CRISPR to identify new targets for bowel cancer tumors when they realized that the gene KMT2A—usually associated with Acute Myeloid Leukemia—promotes bowel cancer. It does this by fuelling uncontrolled growth of the tumor, and encouraging the cancer cells ability to 'self-renew," preventing the tumor from regression or differentiation.

They then trialed two agents that inhibit KMT2A and found that these block bowel cancer growth and self-renewal, with very little damage to normal cells. These inhibitors are very similar to others which are currently in clinical trials to treat leukemia.
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mRNA vaccine yields full protection against malaria in mice
https://medicalxpress.com/news/2021-06- ... laria.html
by Walter Reed Army Institute of Research

Scientists from the Walter Reed Army Institute of Research and Naval Medical Research Center partnered with researchers at the University of Pennsylvania and Acuitas Therapeutics to develop a novel vaccine based on mRNA technology that protects against malaria in animal models, publishing their findings in npj Vaccines.

In 2019, there were an estimated 229 million cases of malaria and 409,000 deaths globally, creating an extraordinary cost in terms of human morbidity, mortality, economic burden, and regional social stability. Worldwide, Plasmodium falciparum is the parasite species which causes the vast majority of deaths. Those at highest risk of severe disease include pregnant women, children and malaria naïve travelers. Malaria countermeasures development has historically been a priority research area for the Department of Defense as the disease remains a top threat to U.S. military forces deployed to endemic regions.
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Yuli Ban
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UMD introduces new CRISPR 3.0 system for highly efficient gene activation in plants
In a study in Nature Plants, Yiping Qi, associate professor of Plant Science at the University of Maryland (UMD), introduces a new and improved CRISPR 3.0 system in plants, focusing on gene activation instead of traditional gene editing. This third generation CRISPR system focuses on multiplexed gene activation, meaning that it can boost the function of multiple genes simultaneously. According to the researchers, this system boasts four to six times the activation capacity of current state-of-the-art CRISPR technology, demonstrating high accuracy and efficiency in up to seven genes at once. While CRISPR is more often known for its gene editing capabilities that can knock out genes that are undesirable, activating genes to gain functionality is essential to creating better plants and crops for the future.
"While my lab has produced systems for simultaneous gene editing [multiplexed editing] before, editing is mostly about generating loss of function to improve the crop," explains Qi. "But if you think about it, that strategy is finite, because there aren't endless genes that you can turn off and actually still gain something valuable. Logically, it is a very limited way to engineer and breed better traits, whereas the plant may have already evolved to have different pathways, defense mechanisms, and traits that just need a boost. Through activation, you can really uplift pathways or enhance existing capacity, even achieve a novel function. Instead of shutting things down, you can take advantage of the functionality already there in the genome and enhance what you know is useful."
And remember my friend, future events such as these will affect you in the future
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Yuli Ban
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He Inherited A Devastating Disease. A CRISPR Gene-Editing Breakthrough Stopped It
Patrick Doherty had always been very active. He trekked the Himalayas and hiked trails in Spain.

But about a year and a half ago, he noticed pins and needles in his fingers and toes. His feet got cold. And then he started getting out of breath any time he walked his dog up the hills of County Donegal in Ireland where he lives.

"I noticed on some of the larger hill climbs I was getting a bit breathless," says Doherty, 65. "So I realized something was wrong."

Doherty found out he had a rare, but devastating inherited disease — known as transthyretin amyloidosis — that had killed his father. A misshapen protein was building up in his body, destroying important tissues, such as nerves in his hands and feet and his heart.

Doherty had watched others get crippled and die difficult deaths from amyloidosis.

"It's terrible prognosis," Doherty says. "This is a condition that deteriorates very rapidly. It's just dreadful."

So Doherty was thrilled when he found out that doctors were testing a new way to try to treat amyloidosis. The approach used a revolutionary gene-editing technique called CRISPR, which allows scientists to make very precise changes in DNA.

"I thought: Fantastic. I jumped at the opportunity," Doherty says.
And remember my friend, future events such as these will affect you in the future
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CRISPR used to treat rare genetic disease in promising phase 1 trial
By Michael Irving
June 27, 2021
https://newatlas.com/medical/crispr-pha ... osis-attr/
Scientists are reporting the first clinical data showing that CRISPR gene editing can be done safely and effectively inside the body. CRISPR was injected directly into the bloodstream of patients with a rare genetic disease, and appeared to work better than current treatments with no serious side effects.

The CRISPR gene-editing tool, which makes precise cut-and-paste edits to the genome inside cells, has shown tremendous promise in treating a wide range of diseases. It’s currently the subject of many human trials, but in almost all of them the actual editing takes place outside the body – cells are removed from the patient, edited and then returned to the body. Another recent study involved injections of CRISPR into the eye to correct a genetic form of blindness, but the results have yet to be published.

Now researchers have released interim data of the first six patients in an ongoing Phase 1 trial conducted by Intellia Therapeutics and Regeneron Pharmaceuticals, marking the first published results of a CRISPR clinical trial involving human gene editing in vivo.

The trial is investigating a CRISPR candidate called NTLA-2001 as a treatment for a disease called transthyretin amyloidosis (ATTR amyloidosis). This rare hereditary disease occurs due to mutations in the TTR gene, which causes a patient to produce misfolded transthyretin (TTR) proteins. These abnormal proteins then build up on nerves and around organs, leading to pain and complications, and can be fatal.
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Sweet success: First precision breeding of sugarcane with CRISPR-Cas9
https://phys.org/news/2021-07-sweet-suc ... -cas9.html
by University of Illinois at Urbana-Champaign

Sugarcane is one of the most productive plants on Earth, providing 80 percent of the sugar and 30 percent of the bioethanol produced worldwide. Its size and efficient use of water and light give it tremendous potential for the production of renewable value-added bioproducts and biofuels.

But the highly complex sugarcane genome poses challenges for conventional breeding, requiring more than a decade of trials for the development of an improved cultivar.

Two recently published innovations by University of Florida researchers at the Department of Energy's Center for Advanced Bioenergy and Bioproducts Innovation (CABBI) demonstrated the first successful precision breeding of sugarcane by using CRISPR/Cas9 genome editing—a far more targeted and efficient way to develop new varieties.
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Gene therapy success offers hope for reversing rare genetic diseases
By Rich Haridy
July 12, 2021
https://newatlas.com/medical/gene-thera ... -children/
A landmark study published in the journal Nature Communications is describing the extraordinarily successful results of a Phase 1 trial testing a targeted gene therapy for children with a rare genetic disease. The research demonstrates a novel method for delivering gene therapies to specific locations deep in the brain and suggests these kinds of genetic treatments could reverse damage in older subjects born with developmental diseases.

The research focused on children born with a very rare genetic disorder called AADC deficiency. The condition involves a single gene mutation that leads to a deficiency in synthesizing key neurotransmitters – dopamine and serotonin. Children born with the disease suffer severe developmental deficits and motor disabilities, often leaving them unable to speak or feed themselves.
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Gene editing 'blocks virus transmission' in human cells
Researchers in Australia said the tool was effective against viral transmissions in lab tests, adding that they hope soon to begin animal trials on the method.

Scientists have used CRISPR gene-editing technology to successfully block the transmission of the SARS-CoV-2 virus in infected human cells, according to research released Tuesday that could pave the way for COVID-19 treatments.

Writing in the journal Nature Communications, researchers in Australia said the tool was effective against viral transmissions in lab tests, adding that they hoped to begin animal trials soon.

CRISPR, which allows scientists to alter DNA sequences and modify gene function, has already shown promise in eliminating the genetic coding that drives the development of children's cancer.

The team in Tuesday's study used an enzyme, CRISPR-Cas13b, that binds to relevant RNA sequences on the novel coronavirus and degrades the genome it needs to replicate inside human cells.

Lead author Sharon Lewin from Australia's Peter Doherty Institute for Infection and Immunity told AFP that the team had designed the CRISPR tool to recognize SARS-CoV-2, the virus responsibly for COVID-19.

"Once the virus is recognized, the CRISPR enzyme is activated and chops up the virus," she said.
https://phys.org/news/2021-07-gene-bloc ... human.html
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RNA modification may protect against liver disease
https://medicalxpress.com/news/2021-07- ... sease.html
by University of California, Los Angeles
A chemical modification that occurs in some RNA molecules as they carry genetic instructions from DNA to cells' protein-making machinery may offer protection against non-alcoholic fatty liver, a condition that results from a build-up of fat in the liver and can lead to advanced liver disease, according to a new study by UCLA researchers.

The study, conducted in mice, also suggests that this modification—known as m6A, in which a methyl group attaches to an RNA chain—may occur at a different rate in females than it does in males, potentially explaining why females tend to have higher fat content in the liver. The researchers found that without the m6A modification, differences in liver fat content between the sexes were reduced dramatically.

In addition, in a preclinical model, the investigators demonstrated that gene therapy can be used to enhance or add modifications to key RNAs to slow down or reduce the severity of liver disease.
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