Crispr Pioneer Is Aiming Towards Brain Diseases

In recent news, a CRISPR pioneer, Feng Zhang, from the Broad Institute of MIT and Harvard has started up his own pharma company. Dubbed as Beam Therapeutics, it has made headlines because Zhang has collected up to USD 222 million for research funds and development. Zhang largely focusing on editing gene structure and has recently released a ground breaking invention that is expected to be able to manipulate RNA in genes.

This may equip researchers and scientists to one find cures for different brain diseases, such as the likes of Alzheimer’s. The system developed by Zhang and his co-workers is commonly known as RNA Editing for Specific C to U Exchange, or RESCUE for short. Keep reading to find out everything to know about this new invention and what we can expect.

1.    How does it work?

In their research journal, Zhang and his colleagues explain exactly how this new technology works. To put it simply, the technology works inside the cells of the body and alters the gene responsible for late-onset Alzheimer’s to a non-pathogenic form. Previously, the method used for this purpose caused the DNA to split at certain points, posing the risk of altering the DNA chains permanently.

Zhang’s invention targets RNA instead of DNA by using a muted version of another enzyme. To get into the particular, ‘RESCUE’ targets on the base chains of RNA called Cytosine. Cytosine can then be converted into Uridine, which can help manipulate the process of protein synthesis or similar processes that have to do with the genetic restructuring. 

2.    The science behind the project

Speaking in terms of brain disease, as aforementioned, RESCUE can alter the genetic structure in RNA to alter the variant that poses a risk of Alzheimer’s to a non-pathogenic state. According to Zhang and his team, this removes the risk of late-onset Alzheimer’s altogether. RESCUE and RNA editing can also be used to reverse certain synthesis processes to promote healing of wounds. RESCUE largely works on the principle of altering RNA and activates certain hormones in the body to help further the process.  

3.    Future Projections

Taking inspiration from Zhang’s model, other companies and research groups have also begun work on similar technologies. For the future, researchers are confident that these cures can be extended to help with other diseases as well, including Parkinson’s and Duchenne Muscular Dystrophy. There is still plenty more work that needs to be done on RESCUE itself before it can be used practically. Zhang has said that clinical trials are still a little way down the road. This is because there is a very specific set of technologies needed to develop RESCUE.

Conclusion   

Even though CRISPR technology and RESCUE has some ground to cover before it can be used on people, it is a step in the right direction when it comes to the fight against brain diseases. However, using advanced programming and enhancing the precision of RNA editing, some promising results can be expected shortly.

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