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Nov 12, 2019

Gene-editing Gets Major Funding

Posted by in categories: biotech/medical, genetics

The program, called Somatic Cell Genome Editing, will be investing $190 million. (2018)


Last year, I wrote about a team of Chinese scientists having received ethical approval to perform a clinical trial of gene-editing. The goal was to test whether gene-editing may be a potential cure for cancer. The technology used for the trial is called CRISPR/Cas9, not exactly a household name. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. Cas9 stands for CRISPR associated protein 9, an RNA-guided DNA endonuclease enzyme. If you read all these words a few times, it can make your head hurt. The topic is complex, but I hope in this post to make it more understandable.

After reading about CRISPR more than a few times, I think I finally get the concept. I may not have this 100% right, but following is what I believe it is about. To imagine what gene-editing is, consider editing of a video. The software shows you each frame of the video. You select a frame you want to edit and display the frame in video editing software. You make the changes to look the way you want the frame to look, and then insert the frame back into the video. For example, the original video may have contained an unneeded “um” or “ah” or “eh” which added no value to the video.

Now, consider the similarity with gene-editing. The human body has T-cells which are an active participant in our immune system. A gene in the T-cells can produce a protein called PD-1 which disables the T-cells’ ability to trigger an immune response to fight cancer. A team of oncologists removed cells from an advanced stage lung cancer patient and edited the cells using CRISPR-Cas9. After editing out the gene which blocks the immune response, the cells were cultured and multiplied and then injected back into the patient.

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