The scientific world is captivated by this revolutionary technologysince it is easier, cheaper and more efficient than previous strategies for modifying DNA.
The findings represent not only another piece of information to understand the molecular mechanisms triggering ALS, but also support the newly identified genes as potential targets for future therapeutics. The accumulation of toxic protein clumps in nerve cells is the underlying cause of several neurodegenerative diseases, including ALS.
It is known that mutations in the C9orf72 gene are the most common genetic cause for ALS.
These errors expand the number of DNA repeats contained within the gene, triggering the disease. This strategy allows researchers to identify genes that help prevent or enhance toxicity.
If, after knocking-out a particular gene, researchers see the repeats of the protein encoded by the mutated C9orf72 gene are no longer toxic, it means the absence of the gene is actually beneficial, making it a potential therapeutic target.
In the next set of experiments, they switched to primary mouse neurons the cells of relevance for ALS and performed the screen all over again, but this time just for the genes shown to have a role in ALS.
This is the first study, researchers believe, to use genome-wide human CRISPR knockout screen to unveil the molecular mechanisms underlying a neurodegenerative disease, in this case ALS.An illustration of the CRISPR-Cas9 gene editing complex from Streptococcus pyogenes.
The Cas9 nuclease protein uses a guide RNA sequence to cut DNA at a complementary site. What is gene editing? First, let's look at exactly what gene editing is.
A gene is made up segments of DNA (deoxyribonucleic acid).
DNA is structured as a double helix, which looks somewhat like a. Gene-editing technology isn’t just for movies like Rampage, which stars Dwayne Johnson blitzing a foot wolf (thus leading Oliver to suggest the film should have been named Wolf Blitzer.
1. Sangamo Therapeutics Sangamo Therapeutics is by far the biggest winner among gene-editing biotech stocks right now. Its share price has nearly quintupled over the past 12 months.
CRISPR (/ ˈ k r ɪ s p ər /) (clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found within the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments from viruses that have previously infected the prokaryote and are used to detect and destroy DNA from similar viruses during subsequent infections.
With CRISPR/Cas9 and other genome-editing technologies, successful somatic and germline genome editing are becoming feasible. To respond, an American Society of Human Genetics (ASHG) workgroup developed this position statement, which was approved by the ASHG Board in March