A False Reason for Unwarranted Selling of CRISPR/Cas9 Gene Editing Shares
CRISPRs are bacterial immune systems that contain many defense enzymes such as the Cas9 “molecular scissors,” which scientists have engineered as a powerful DNA-targeting gene-editing tool and created companies specialized in CRISPR gene editing tools.
The outstanding revolutionary news, which is wrongly interpreted by Wall Street, which led to a recent selling of the shares of CRISPR gene editing firms has come from the Salk Institute. The remarkable news announced the success of the Institute’s Scientists in discovering enzymes that target RNA not DNA, which is targeted by the CRISPR/Cas9 gene editing.
Indeed, the Salk Institute’s discovered the Cas13d enzyme.
Like the Cas9 family, Cas13d enzymes originate from different bacterial species, hence would vary in their activity. For that reason, the Institute’s scientists ran a screen to identify the best version for use in human cells. That version turned out to be from the gut bacterium Ruminococcus flavefaciens XPD3002, which led them to name their tool CasRx.
This discovery was essential as many genetic diseases are caused by RNA problems, not just by DNA problems. The RNA problems include a possible RNA imbalance relative to other RNAs. The balance is critical for the healthy function of the body. Also, RNA can be spliced in various ways to make different proteins. Problems with splicing can lead to diseases such as spinal muscular atrophy, atypical cystic fibrosis and frontotemporal dementia (FTD).
So targeting injurious RNAs or RNAs that results from improper splicing could have a life-changing impact for people with these types of devastating diseases.
CasRx, the Salk Institute’s press release stated, is offering researchers a powerful way to develop new gene therapies as well as investigate fundamental biological functions.
Correcting a Protein Imbalance in Dementia Patient
As a matter of fact, the scientists used CasRx, to correct a protein imbalance in cells from dementia caused by frontotemporal degeneration (FTD) — a disease process that results in progressive damage to the temporal and/or frontal lobes of the brain. The approach restored the balance to the cells’ proteins. CasRx was 80 percent effective in rebalancing the levels of tau protein to healthy levels.
The Salk Institute’s work appeared in the Journal Cell on March 15, 2018.
This fantastic news does not in any way suggest bad news for the firms specialized in CRISPR/Cas9 gene editing technology. On the contrary, it presented these firms with opportunities to use the new Cas13d enzyme for the discovery of genetic diseases and treating them as referred to by the Salk Institute.
One of the contributing researchers, Patrick Hsu, a Helmsley-Salk Fellow and senior author of the new paper explained, “Bioengineers are like nature’s detectives, searching for clues in patterns of DNA to help solve the mysteries of genetic diseases. CRISPR has revolutionized genome engineering, and we wanted to expand the toolbox from DNA to RNA.”
To expand is what the researcher said, not replace CRISPR/Cas9 gene editing, which is considered a super breakthrough.
It is evident that the most beneficiaries from the advancement in CRISPR gene editing technologies are the people with intractable life-threatening genetic diseases.
Will be also benefiting and hailed are the firms that are specialized in CRISP gene editing technology, which continues to be improved, becoming more valuable than any other technology that deals with the discovery, understanding, and treatment of genetic diseases.
These firms comprise: Crispr (CRSP), Intellia (NTLA) and Editas (EDIT), which are the ones being targeted for an unwarranted selloff based on misinterpreting good news.
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