In the News
Ayal Hendel Talks About CRISPR in Bubble Boy Disease
Dr. Ayal Hendel, a genome-editing pioneer and group leader at Bar Ilan University in Israel, was one of the first researchers to use modified synthetic sgRNA to edit primary cells with CRISPR. In this episode, Dr. Hendel shares his perspectives on the recent CRISPR advancements and his work on bubble boy disease.
Gene Replacement Emerges as New Immunodeficiency Therapy
Two research groups are independently pioneering a groundbreaking gene-editing treatment for severe immunodeficiencies, potentially transforming the lives of patients. This innovative approach targets the critical RAG1 and RAG2 genes, promising a permanent solution that could surpass traditional bone marrow transplants.
Ayal Hendel Talks About CRISPR in Bubble Boy Disease
Dr. Ayal Hendel, a genome-editing pioneer and group leader at Bar Ilan University in Israel, was one of the first researchers to use modified synthetic sgRNA to edit primary cells with CRISPR. In this episode, Dr. Hendel shares his perspectives on the recent CRISPR advancements and his work on bubble boy disease.
CRISPECTOR Accurately Detects Translocations and Off-Target Activity
For the first time, adverse translocation events can be detected in multiplex PCR experiments after CRISPR editing. A new software tool, CRISPECTOR, efficiently separates signal from noise and can detect, statistically evaluate, and quantify many kinds of off-target genome-editing activities.
CRISPECTOR: A Better Tool for Measuring CRISPR-Cas-induced Adverse Activity
Off-target activity can lead to translocations, unusual rearrangement of chromosomes, as well as to other adverse genomic modifications. These, in turn, can have detrimental consequences such as cancer and other pathologies. It is therefore important to detect such potential events in advance. Our goal in this research was to develop a software tool that accurately and reliably quantifies CRISPR-Cas adverse off-target effects, including translocation events, that occur in an editing experiment.
A CRISPECTOR calls with fresh insight to gene editing errors
An Israeli research team claims new software can detect unintended consequences of gene editing. Here, Dermot Martin unpacks the research that applies statistical modelling to determine and quantify editing activity. The new technology promises to identify and report unintended mutations that might currently go undetected during CRISPR Cas 9 gene editing of early stage human embryos.
New Israeli tech gives accuracy boost to microscopic scissors for DNA editing
Israeli scientists have developed a “quality assurance” method for genome editing, which they say has the power to make it easier to translate DNA technologies from theory to treatments.
The CRISPR genome editing system works by cutting DNA with microscopic “scissors” and fixing it. The method is deployed to address some genetic disorders, and there are huge efforts to expand its use.
But one of the teething troubles is accuracy.
Israeli researchers are developing a CRISPR gene editing therapy for SCID, interview with Ayal Hendel
CRISPR genome editing marches forward as a curative therapy for genetic disorders. Here lead scientist Ayal Hendel talks about how he is developing a CRISPR therapy that can be used to restore a functional immune system in infants with severe combined immune deficiency or SCID. He looks forward to seeing the strategy through to the first clinical trials.
The elegant parallel of using CRISPR to understand disease mechanisms
CRISPR is the buzzword of the moment in the drug discovery industry – mainly due to its potential to correct disease-causing mutations. However, those using the technology need to be mindful that it is used responsibly, and possible risks are considered before use. Mark Behlke discusses the potential of CRISPR in R&D and the challenges that it presents for researchers.
CRISPR marches forward:
Stanford scientists optimize use in human blood cells
The CRISPR news just keeps coming. As we've described here before, CRISPR is a breakthrough way of editing the genome of many organisms, including humans— a kind of biological cut-and-paste function that is already transforming scientific and clinical research. However, there are still some significant scientific hurdles that exist when attempting to use the technique in cells directly isolated from human patients (these are called primary cells) rather than human cell lines grown for long periods of time in the laboratory setting.
Policing the editor:
Stanford scientists devise way to monitor CRISPR effectiveness
As a writer, I think a lot about editing. Will this sentence work here? Maybe I should change this word. Argh - a typo! But I’m not alone. Biologists also appreciate the power of editing, particularly when it comes to modifying genes in cells or organisms.
