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An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials.
(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.
Founded by University of California, Berkeley scientists, Rewrite is studying ways to expand the reach of gene editing beyond limitations of current technologies, including Intellia's.
Moonwalk Biosciences, the latest biotech cofounded by the gene editing scientist, joins other startups aiming to alter gene expression without changing DNA.
Furthermore, researchers found that one of the genes turned on by these BET proteins is ACE2, the same protein that the virus depends on to get into cells. In the latest study, Gladstone graduate student Irene Chen and the team found that the BET proteins switch on genes that block viruses, in SARS-CoV-2 infected cells.
The US Food and Drug Administration (FDA) has granted approval for CSL Behring’s adeno-associated virus vector-based gene therapy, Hemgenix (etranacogene dezaparvovec), to treat haemophilia B (congenital Factor IX deficiency) in adult patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
For decades, scientists have tried unsuccessfully to target the KRAS gene, which is often mutated in lung, colon and pancreatic cancers. Lumakras is the first drug proven effective.
Through a new deal, Roche has exclusive rights to Sangamo molecules designed to repress the gene that makes “tau,” a protein many scientists view as a main driver of Alzheimer’s.
Once the specialty of a few select drugmakers, CRISPR gene editing is now an essential technology for a growing group of biotechs, many led by former students of the field's pioneering scientists.
Sarepta has not provided “unambiguous evidence” its treatment can help patients, agency scientists wrote in documents released ahead of a crucial Friday advisory committee meeting.
A federal patent board ruled Broad Institute scientists were first to a key gene editing advance, weakening the patent position of Intellia and CRISPR Therapeutics, which hold licenses through the University of California.
But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space. A new frontier in cancer research.
Turning off a gene early in mouse development led researchers to end up with an accidental six-legged embryonic mammal. This strange result took the spinal cord research of developmental biologists Anastasiia Lozovska and Moisés Mallo and their colleagues at Portugal’s Gulbenkian Science Institute in a new direction.
Elsewhere, AskBio advanced a gene therapy and Pfizer hired a scientist well versed in GLP-1 science. The Swiss pharma will base cardiometabolic drug research at the new Allston site.
Scientists at Pretzel believe fixing mutated mitochondrial DNA with a mix of small molecule therapies and gene editing could be key to solving a number of hard-to-treat diseases.
A previously unknown mechanism for inactivating genes that suppresses tumor formation helps explain why cancer risk is associated with an unhealthy diet or unmanaged metabolic conditions like diabetes.
Forward genetics pinpoints gene linked to ASD involving severe speech impairment and opens door to search for more mutations, future treatments Credit: Photo taken by Brian Coats for UT Southwestern Medical Center DALLAS – Dec.
Scientists have identified more than 1,500 genetic differences between migratory and non-migratory hoverflies. Credit: Will Hawkes Scientists have identified more than 1,500 genetic differences between migratory and non-migratory hoverflies.
The last time this summit convened in 2018, the world was shocked to hear a scientist had created the first gene-edited babies. He was condemned, but gene-editing has continued, with some success. Image credit: Anthony Wallace/AFP via Getty Images)
An anti-aging gene discovered in a population of centenarians has been shown to rewind the heart's biological age by 10 years. The breakthrough, published in Cardiovascular Research and led by scientists at the University of Bristol and the MultiMedica Group in Italy, offers a potential target for patients with heart failure.
Credit: Professor Jane Farrar and Dr Daniel Maloney, Trinity College Dublin Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision and affects thousands of people across the globe.
Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.
Promising news in the effort to develop drugs to treat obesity: University of Virginia scientists have identified 14 genes that can cause and three that can prevent weight gain. The findings pave the way for treatments to combat a health problem that affects more than 40% of American adults.
Scientists are starting to understand the precise workings of a type of gene that, unlike other genes, does not code for proteins – the building blocks of life.
Scientists at Université de Montréal, McGill University and its affiliated Montreal Children's Hospital have made a promising breakthrough in understanding the origins of mirror movement disorder, a rare inherited neurological disorder.
Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham. Continue to STAT+ to read the full story…
Mutations of a gene called Foxp2 have been linked to a type of speech disorder called apraxia that makes it difficult to produce sequences of sound. A new study from MIT and National Yang Ming Chiao Tung University sheds light on how this gene controls the ability to produce speech.
Credit: David Jandzik New University of Colorado Boulder-led research finds that the traits that make vertebrates distinct from invertebrates were made possible by the emergence of a new set of genes 500 million years ago, documenting an important episode in evolution where new genes played a significant role in the evolution of novel traits in […]. (..)
QUT and Dutch scientists have identified two new genes involved in hemiplegic migraine, a rare, debilitating subtype of migraine that causes weakness along one side of the body during the aura phase.
Scientists at UMass Chan Medical School have developed a technology to deliver gene therapy directly to lung tissue through intranasal administration, a development that could potentially create a new class of treatments for lung disease.
In a new study, scientists combined strategies to deliver energy-disrupting gene therapy using nanoparticles manufactured to zero in only on cancer cells. Researchers are shining a light on cancer cells' energy centersliterallyto damage these power sources and trigger widespread cancer cell death.
New genetic sensors, developed by scientists at University of Warwick and Keele University, could function as a lab test device and even as a live monitoring system inside living cells The innovative system can detect when a specific gene in a cell is active – instead of only detecting its presence The technology is based […].
Mutations in our genes can lead to severe problems, like colon or liver cancer. Mutations in the same genes can lead to different subtypes of tumors in different people. Currently, scientists don't have a good way to produce such tumor subtypes for study in the lab. But cancer is very complex.
Credit: UT Southwestern Medical Center DALLAS – April 8, 2021 – New research has uncovered a surprising role for so-called “jumping” genes that are a source of genetic mutations responsible for a number of human diseases.
Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.
Merck and Synplogen have signed a non-binding Memorandum of Understanding (MoU) to expedite the development and manufacturing of viral vector-based gene therapy applications. The firms intend to merge their expertise to provide simplified viral vector gene therapy development, production and testing in Japan. By Cytiva Thematic.
Scientists working with an experimental mouse model at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) have identified the essential role of the gene GPR126 in the development of the placenta during pregnancy.
Gene editing technology has been used to pinpoint new molecular targets for treating an aggressive form of leukemia in adults. November 24, 2021 Gene editing technology has been used to pinpoint new molecular targets for treating an aggressive form of leukemia in adults. Hokkaido University scientists have identified new […].
Wilmington, DE, (MARCH 21, 2022) – A new study from scientists at ChristianaCare’s Gene Editing Institute is advancing the safety and efficacy of using CRISPR gene editing in patient treatments by demonstrating how to identify and evaluate the broad-based biological impact of gene editing on targeted tissues, where the edits are designed to fully disable (..)
The next COVID pandemic could be prevented by using a gene drive to preemptively edit the genome of bats to prevent them from becoming hosts for coronaviruses, according to a proposal by scientists from Israel’s Interdisciplinary Center Herzelia and the National Institutes of Health.
Researchers at the National Institutes of Health have successfully identified differences in gene activity in the brains of people with attention deficit hyperactivity disorder (ADHD).
SAN FRANCISCO, CA—Using new technologies to study thousands of genes simultaneously within immune cells, researchers at Gladstone Institutes, UC San Francisco (UCSF), and Stanford School of Medicine have created the most detailed map yet of how complex networks of genes function together.
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