CRISPR & Genetic Engineering News and Discussions

Vakanai
Posts: 313
Joined: Thu Apr 28, 2022 10:23 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by Vakanai »

Xyls wrote: Wed Jul 13, 2022 2:07 pm Verve Therapeutics begins human tests of first ‘base editor,’ aiming at heart disease

https://www.statnews.com/2022/07/12/ver ... se-editor/
Somewhere in New Zealand, the first patient ever has been dosed with a kind of gene-editing treatment known as a base editor, a newer way of utilizing CRISPR for gene editing. The company studying the treatment, Verve Therapeutics, announced the news Tuesday.

The treatment is aimed at a relatively common form of high cholesterol that affects millions of people, a very different population than those normally treated with gene therapies. Eventually, Verve hopes that the treatment might be offered to people who have recently suffered heart attacks, or for other more common diseases.
Or you know, maybe offer it to people at risk of heart attack before they have a freaking heart attack?
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

New DNA repair-kit successfully fixes hereditary disease in patient-derived cells
https://medicalxpress.com/news/2022-07- ... sease.html
by University of Bristol
Genetic mutations which cause a debilitating hereditary kidney disease affecting children and young adults have been fixed in patient-derived kidney cells using a potentially game-changing DNA repair-kit. The advance, developed by University of Bristol scientists, is published in Nucleic Acids Research.

In this new study, the international team describe how they created a DNA repair vehicle to genetically fix faulty podocin, a common genetic cause of inheritable Steroid Resistant Nephrotic Syndrome (SRNS).

Podocin is a protein normally located on the surface of specialized kidney cells and essential for kidney function. Faulty podocin, however, remains stuck inside the cell and never makes it to the surface, terminally damaging the podocytes. Since the disease cannot be cured with medications, gene therapy which repairs the genetic mutations causing the faulty podocin offers hope for patients.
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Researchers add second copy of gene to give rice a 40% yield boost

by Bob Yirka , Phys.org
https://phys.org/news/2022-08-gene-rice ... boost.html
A team of researchers affiliated with several institutions in China, working with a colleague from Germany, has boosted the yield of rice by 40% by giving test plants a second copy of a certain gene. In their paper published in the journal Science, the group describes their work in improving rice yields to meet growing food demands in light of a continuing rise in global population. Steven Kelly with the University of Oxford, has published a Perspectives piece in the same journal issue outlining the work done by the team in China.

As the world's population continues to grow, scientists around the world are looking for ways to get more food out of the land available for use in growing crops. In this new effort, the researchers looked at ways to improve rice yields by genetically altering DNA to coax individual plants to produce more grains of rice.
User avatar
caltrek
Posts: 6474
Joined: Mon May 17, 2021 1:17 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by caltrek »

CRISPR-based Technology Targets Global Crop Pest
August 18, 2022

Introduction:
(EurekAlert) Applying new CRISPR-based technology to a broad agricultural need, researchers at the University of California San Diego have set their aims on a worldwide pest known to decimate valuable food crops.

Nikolay Kandul, Omar Akbari and their colleagues first demonstrated the precision-guided sterile insect technique, or pgSIT, in Drosophila melanogaster, the common fruit fly, in 2019. The technology, later adapted to mosquitoes, uses programmable CRISPR techniques to edit key genes that control sex determination and fertility. Under the new system, pgSIT-developed insect eggs are deployed into a targeted population and only sterile males hatch, resulting in a fertility dead end for that species.

Kandul, Akbari and their colleagues have now adapted the technology for use in Drosophila suzukii , an invasive fruit fly (also known as the spotted-wing drosophila) responsible for millions of dollars in crop damage. The advancement is described in the journal GEN Biotechnology.

“It’s a safe, evolutionary stable system,” said Akbari, a professor in the School of Biological Sciences’ Department of Cell and Developmental Biology. “Also, the system does not lead to uncontrolled spread nor does it persist in the environment—both important safety features that will help it gain approvals for use.”

D. suzukii flies have invaded many parts of the world and caused widespread agricultural and economic damage to several crops, including apples, cherries, raspberries, blueberries, strawberries, peaches, grapes, olives and tomatoes.
Read more here: https://www.eurekalert.org/news-releases/961913
Don't mourn, organize.

-Joe Hill
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

A step toward the creation of materials controlled by artificial genes
https://phys.org/news/2022-08-creation- ... genes.html
by Samuel Schaffter and Gina Wadas, Johns Hopkins University
Our bodies' genes work together to regulate how our cells behave. For example, if you skin your knee, your genes use a chemical messaging system to direct an army of cells to heal the abrasion. If scientists could create artificial genes that could carry out the same functions but operate inside materials rather than organisms, a wide variety of new diagnostic, self-healing materials would be possible.

A team led by Johns Hopkins engineer Rebecca Schulman is laying the foundation for that work by engineering synthetic chemical systems that can emulate the complex behaviors of natural gene networks. Their work recently appeared in Nature Chemistry.

"Cells use genes to decide how to move, grow, and act. The ability to make simple 'genes' that could make decisions on their own could lead to better diagnostics or therapeutics, or even provide ways to build new types of soft material robots that are controlled by chemistry instead of electronics," said Schulman, who is an associate professor of chemical and biomolecular engineering and associate researcher at the Whiting School of Engineering's Institute for NanoBioTechnology.

The human body comprises about 25,000 genes, and the chemical interactions that these genes use to regulate cells have many steps and moving parts. Researchers have learned that they don't need to meticulously recreate every one of these natural biological steps to create synthetic gene analogs capable of carrying out the same functions. To improve and better predict the behavior of gene analogs, Schulman and her team created a molecular tool kit which includes genelets (very small genes whose functions can vary, depending on instructions), and simplified mathematical models that predict how the genelets will behave.
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Microscopy reveals mechanism behind new CRISPR tool
https://phys.org/news/2022-08-microscop ... -tool.html
by Cornell University
New research from Cornell offers insights into a line of CRISPR systems, which could lead to promising antiviral and tissue engineering tools in animal and plants.

The research by Ailong Ke, the Robert J. Appel Professor of molecular biology and genetics in the College of Arts and Sciences, and Stan J.J. Brouns at Delft University of Technology in the Netherlands, focuses on a newly discovered CRISPR RNA-guided Caspase system, otherwise known as Craspase.

CRISPR-Cas systems are RNA-guided nucleases in bacteria that cleave viral DNA or RNA targets in precise locations to enable powerful genome editing applications. Caspases are a family of proteases that control programmed cell death in animals, including humans. A recent finding that caspase-like proteins could associate with CRISPR-Cas electrified the scientific community. Such CRISPR-guided caspases were given a new name, Craspase.

"On one hand, this association was totally unexpected and points to novel modes of antiviral action in bacteria," Ke said. "On the other hand, we could use a system like this to develop many biotech and therapeutic applications, if we understand all the gizmos inside this machinery."
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

A cellular engineering breakthrough: High-yield CRISPR without viral vectors
https://phys.org/news/2022-08-cellular- ... viral.html
by Sarah Williams, Gladstone Institutes

A new variation of the CRISPR-Cas9 gene editing system makes it easier to re-engineer massive quantities of cells for therapeutic applications. The approach, developed at Gladstone Institutes and UC San Francisco (UCSF), lets scientists introduce especially long DNA sequences to precise locations in the genomes of cells at remarkably high efficiencies without the viral delivery systems that have traditionally been used to carry DNA into cells.

"One of our goals for many years has been to put lengthy DNA instructions into a targeted site in the genome in a way that doesn't depend on viral vectors," says Alex Marson, MD, Ph.D., director of the Gladstone-UCSF Institute of Genomic Immunology and senior author of the new study. "This is a huge step toward the next generation of safe and effective cell therapies."

In the new paper published in the journal Nature Biotechnology, Marson and his colleagues not only describe the technology but show how it can be used to generate CAR-T cells with the potential to fight multiple myeloma, a blood cancer, as well as to rewrite gene sequences where mutations can lead to rare inherited immune diseases.
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Stem cell-gene therapy shows promise in ALS safety trial

by Cedars-Sinai Medical Center
https://medicalxpress.com/news/2022-09- ... html[quote]

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Lou Gehrig's disease.

In the first trial of its kind, the Cedars-Sinai team showed that delivery of this combined treatment is safe in humans. The findings were reported today in the peer-reviewed journal Nature Medicine.

"Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that can't otherwise get through the blood-brain barrier," said senior and corresponding author Clive Svendsen, Ph.D., professor of Biomedical Sciences and Medicine and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute. "We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS."

Aimed at preserving leg function in patients with ALS, the engineered cells are potentially a powerful therapeutic option for this disease that causes progressive muscle paralysis, robbing people of their ability to move, speak and breathe. [/quote]
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Researchers develop gene therapy for rare ciliopathy
https://medicalxpress.com/news/2022-09- ... pathy.html
by National Eye Institute
Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood. Using patient-derived retina organoids (also known as retinas-in-a-dish), the researchers discovered that a type of LCA caused by mutations in the NPHP5 (also called IQCB1) gene leads to severe defects in the primary cilium, a structure found in nearly all cells of the body. The findings not only shed light on the function of NPHP5 protein in the primary cilium, but also led to a potential treatment for this blinding condition. NEI is part of the National Institutes of Health.

"It's so sad to see little kids going blind from early onset LCA. NPHP5 deficiency causes early blindness in its milder form, and in more severe forms, many patients also exhibit kidney disease along with retinal degeneration," said the study's lead investigator, Anand Swaroop, Ph.D., senior investigator at the NEI Neurobiology Neurodegeneration and Repair Laboratory. "We've designed a gene therapy approach that could help prevent blindness in children with this disease and one that, with additional research, could perhaps even help treat other effects of the disease."

LCA is a rare genetic disease that leads to degeneration of the light-sensing retina at the back of the eye. Defects in at least 25 different genes can cause LCA. While there is an available gene therapy treatment for one form of LCA, all other forms of the disease have no treatment. The type of LCA caused by mutations in NPHP5 is relatively rare. It causes blindness in all cases, and in many cases it can also lead to failure of the kidneys, a condition called Senior-Løken Syndrome.
weatheriscool
Posts: 12727
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

New approach more than doubles stem cell editing efficiency, researchers report
https://phys.org/news/2022-09-approach- ... iency.html
by Mary Fetzer, Pennsylvania State University
A Penn State-led team of interdisciplinary researchers has developed techniques to improve the efficiency of CRISPR-Cas9, the genome editing technique that earned the Nobel Prize in 2020. While CRISPR-Cas9 is faster, less expensive and more accurate than other gene-editing methods, according to project leader Xiaojun "Lance" Lian, associate professor of biomedical engineering and biology at Penn State, the technology has limitations—especially in applications to improve human health.

The researchers developed a more efficient and accessible process to apply CRISPR-Cas9 systems in human pluripotent stem cells (hPSCs), derived from federally approved stem cell lines, which Lian said could greatly advance diagnostics and treatments for genetic disorders. The approach was published Sept. 7 in Cell Reports Methods.

CRISPR-Cas9, which stands for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, gives scientists the ability to target precise locations of genetic code to change DNA, providing opportunities to create new diagnostic tools and potentially correct mutations to treat genetic causes of disease.

"The human genome is enormous, and CRISPR-Cas9 makes it possible for scientists to find and target a mutated gene for the purpose of studying it," Lian said.

CRISPR uses a disc of genetic material, known as plasmid DNA, to deliver guided ribonucleic acid (RNA) that positions the Cas9 enzyme at the precise location of the target gene. When the DNA is located, Cas9 binds to it and cuts it out, allowing other DNA to repair the cut. Researchers can then see how the removal changes the gene's expression. But there are delivery and editing efficiency problems with current DNA-based CRISPR methods, according to Lian.
Post Reply