The CRISPR/ Cas9 Gene editing technology succeeded to remove the mutations in the cells grown at lab. But with the change in time and the development in this technology, CRISPR is now used to treat a fatal disorder. Researchers injected the drug into the blood of human being diagnosed with the disease. In addition, the liver has nearly ceased the production of toxic protein which eventually caused fatal nerve and heart disorder.
It is not clear whether the symptoms of the disease Transthyretin Amyloidosis can be cured completely. Even then CRISPR provided a ray of hope to the suffering ones as well as the medical team. Molecules of Transthyretin (TTR) protein folds into wrong shapes. Then these cluster together to form fibres which eventually interferes with the functioning of an organ. In the case of Transthyretin Amyloidosis, TTR proteins build up on nerves and heart causing pain, numbness and results in heart disease. The medicine Patisiran, used to stabilise this rare condition.
Thereafter the researches at Veteran Biotech Regeneron Pharmaceuticals and the startup Intellia Therapeutics thought of injectable CRISPR treatment against the disease. Two women and four men of the age group 46 to 64 with Transthyretin Amyloidosis chose for the trial treatment. They were injected with lipid particle that carried two RNA’s. They are:
1) mRNA (messenger RNA): It encodes a protein Cas and CRISPR component which cuts DNA.
2) Guide RNA: It directs CRISPR to the gene for TTR. Cas cuts the unwanted portion and then the repair machinery of the DNA will start the healing process.
The team observed 80% to 96% decrease in TTR levels in three men who were given the higher of two doses of the treatment. This was a result which was far better from the average of 81% with Patisiran. In accordance with David Adams (Neurologist at the University of Paris-Saclay), this treatment can be potentially the first curative one for this life threatening disease and disabling of hereditary.
Well, are you thinking about the side effects? It would take time to understand whether the symptoms shown by the patients got reduced. One of the major issues is that if CRISPR cut wrong DNA location it may lead to cancer or some other problems.
Also the use of lipid encased mRNA approach is safer than virus for the transport of the genetic instruction. This information is for encoding an editing protein and in guiding mRNA into cells. The treatment using CRISPR leads the way in curing other liver diseases. Either CRISPR can knock out the mutated gene or modify it with the use of DNA template.
According to Jennifer Doudna, Nobel Laureate 2020 who developed CRISPR from bacterial immune system(co-founder of Intellia Therapeutics), this study is a critical first step in enabling repair and inactivation of the disease causing gene anywhere in the body.
Hope that CRISPR technology can wipe out all the disorders which may occur anywhere in the human body.
https://www.sciencemag.org/news/2021/06/crispr-injected-blood-treats-genetic-disease-first-time
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https://shasthrasnehi.com/optogenetics-helps-in-neuroprosthetic/
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