CRISPR Therapeutics applied for permission from European regulators to begin testing their newest, most advanced gene editing product, CTX001, on human patients.
Scientists from CRISPR Therapeutics plan to use their newest advancement to try treating patients suffering from beta thalassaemia, a blood disease where the body does not produce enough healthy red blood cells. Patients who have the most severe cases of this inherited blood disease need to have frequent transfusions in order to prevent death.
The genes that cause this disease will be edited out using an advanced version of the CRISPR-Cas9 technology.
CRISPR-Cas9 technology can be thought of having two parts. The first part is Cas9, a protein that acts like a pair of molecular scissors that is able to cut strands of DNA in order to remove undesirable or mutated genetic material and create space for functioning genes to be inserted. CRISPR acts as a genetic GPS to guide the scissors to the exact location.
Scientist first identify an effective DNA strand to be cut out and modified. They then create guide RNA that has the same genomic sequence as the defective DNA. This is combined in a cell with the Cas9 protein. The guide RNA finds the matching genomic sequence as this happens.
Then the Cas9 cuts the strand, making a break in the DNA helix while cells detect and repair the broken DNA. A healthy strand of DNA is inserted at the cut site and enzymes repair it. The cell repair systems can use a piece of complementary DNA, called the template, in order for scientists to add beneficial changes to the template, like correcting a disease-causing mutation.
Despite the Cas9 enzyme being incredibly precise with undetectable mistakes, it is hard to say what experimenting on humans could result in in the long run.
Freshman Justin Siochi says, “The obvious answer would be to do anything to help and treat people who are affected, but risk of error and medical problems as well as the amount of control a person would have over this research attaches problems to what seems to be an ideal situation.”
Ever since the release of the technology, there has been debate over the ethics surrounding it. CRISPR can target any genes, even those coding for a person’s cosmetics. When tampering in those genes begins, a scary and tedious line develops. Without regulations, this technology could be abused for more trivial reasons, rather than to simply help with diseases and sickness.
“I feel like it is an amazing advancement in technology and, when implemented right, it can be good. But I feel like it could become a cosmetic based industry strictly for money making, and, with that, it would have lost its main purpose,” says freshman Christian Cipolletta.
If these trials are successful, CRISPR Therapeutics says that they plan to study the technique in humans with a variety of diseases, including cancer, cystic fibrosis, hemophilia, and more.
This advancement is a the key to curing diseases and beginning to ensure happy and healthy lives for those who suffer unjustly.
Where should the line be drawn between ethically just and trivial matters for gene editing?
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