Abstract BACKGROUND Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based
Abstract The application of RNA interference (RNAi) to mammalian cells has provided the means to perform phenotypic screens to determine the functions of genes. Although
Abstract The expanding applications of nonviral genomic medicines in the lung remain restricted by delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen
Abstract Class 2 CRISPR effectors Cas9 and Cas12 may have evolved from nucleases in IS200/IS605 transposons. IscB is about 2/5 the size of Cas9 but
AbstractWith the development of CRISPR/Cas9-mediated gene editing technologies, correction of diseasecausing mutations has become possible. However, current gene correction strategies preclude mutationrepair in post-mitotic cells
One mouse is hunched over, graying, and barely moves at 7 months old. Others, at 11 months, have sleek black coats and run around. The
In a biological beating of swords into plowshares, researchers have converted a bacterial toxin into a genome editing tool that, for the first time, can
Abstract The recent advancements in CRISPR/Cas9 engineering have resulted in the development of more targeted and potentially safer gene therapies. The challenge in the cancer
Amyotrophic lateral sclerosis (ALS) is a debilitating and fatal disorder that can be caused by mutations in the superoxide dismutase 1 (SOD1) gene. Although ALS
Preliminary research suggests that using CRISPR to treat cancer is safe in humans and could become a feasible therapeutic method in the future, although its
A new gene editing technique called prime editing, tested in human and mouse cells, rewrites DNA by only cutting a single strand to add, remove,
A study published in Nature Medicine that found an increased mortality rate in people with an HIV-preventing gene variant has a major flaw, according to
Sientists have used CRISPR technology to edit out a lung-disease–causing gene while mice were in utero, rescuing the function of the lung and not affecting
An advisory panel to the World Health Organization has called for the creation of a global registry to monitor gene-editing research in humans, the organization
There should be a global moratorium on editing heritable DNA from humans, scientists and bioethicists from seven countries write today (March 13) in a Nature
In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results from a clinical trial, shared
In 2018, scientists rode the activist momentum of 2017 to step out of the lab and into the public sphere: There was an unprecedented surge
The world’s first gene-edited babies were announced early this week not in a scientific publication or at a conference, but in news reports. He Jiankui,
Japan has issued draft guidelines that allow the use of gene-editing tools in human embryos. The proposal was released by an expert panel representing the
A therapy that edits genes directly in the human body might be safe, suggest early findings from the first trial to test the approach. Researchers
Researchers in the US and Europe have used two gene-editing approaches to correct mitochondrial mutations in live mice. The results, published yesterday (September 24) in
Newly identified anti-CRISPR proteins block Cas12a, a cousin of the Cas9 nuclease, both of which are widely used in genome editing, two teams of researchers
CRISPR gene editing can create large numbers of unwanted DNA changes that may go undetected, researchers report today (July 16) in Nature Biotechnology. The findings
Doctors at the University of Pennsylvania posted plans for a clinical trial, which will combat cancer using human immune cells reprogrammed with CRISPR, on ClinicalTrials.gov
Last Thursday (December 7), CRISPR Therapeutics submitted an application to European regulatory authorities seeking permission to begin clinical trials for CTX001, an investigational CRISPR treatment
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