The field of medicine is advancing and evolving at the speed of light. Genetic engineering is a field that has seen the daylight of progress and research in the past couple of years. Scientists of today are in a continuous struggle to decode the role of genes played in the occurrence of prevalent diseases. The multifactorial struggle is based on diagnosing the disease and treating it using gene therapy.
In this maneuver of peeping into the windows of DNA, geneticists found an extremely useful gene-editing tool i.e. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). In the year 2020, scientists combined CRISPR with special CRISPR-associated proteins i.e. Cas9 to achieve success in fields that they suffered in previously.
Who Is Using CRISPR Currently?
CRISPR is, in fact, a defense mechanism sequence found in the DNA of bacteria that they use to save themselves against attack by viruses. This palindromic repeat sequence is paired with a CRISPR-associated enzyme to cleave DNA and edit genetic makeup in less time and cost as compared to the prevalent gene editing mechanisms.
So far the CRISPR technology is being used in clinical trials. There is a wide spectrum of areas where CRISPR is being tested. Ranging from agricultural output to medical treatments, CRISPR has shown promising results.
Though the technique is limited to clinical trials, for now, we can expect CRISPR to become the mainstay treatment for the following conditions:
CRISPR/Cas9 technique has been used in clinical trials to manage cancer patients. Prostate and breast cancer patients have shown positive results with the use of the CRISPR system.
This technology of gene editing can be used to diagnose cancer (in a cost-effective way) and boost the host immune system against cancer cells. This type of gene editing can also be used in the future to directly causing mutation in the cancerous cells (as indicated by animal models).
Blood dyscrasias and other related disorders have a genetic base in most cases. It is in this field that doctors are baffled and aggrieved at the same time. Grieved because there is no cure. Genetic engineers are in a struggle to formulate a way of curing sickle cell anemia and beta thalassemias.
The best way to estimate the time required for a particular cure to become public is to check how many clinical trials are being conducted for it. The year 2020 has seen plenty of clinical trials of CRISPR for treating blood disorders.
Back in 2016, CRISPR/Cas9 technology was tested as a new tool in basic hematology. It is needless to say that the technique proved its nickel. The use of the CRISPR system for curing anemias, hemoglobin abnormalities, coagulation disorders can change the whole dimension of hematological treatments. (1)
By December 2020 we started getting articles claiming that tweaking genes with CRISPR can fix blood disorders. (2)
This indicates that we are near to get CRISPR for blood disorder treatment.
Hereditary blindness has had no definitive cure up till now. But, we can expect this to change. A person with genetic blindness has become the first one alive to receive CRISPR-Cas9 gene therapy directly into the body. (3)
Hereditary blindness has had no definitive cure up till now. But, we can expect this to change. A person with
Where the untimely advent of COVID-19 has stricken the world, CRISPR technology has flourished manifolds. Everything related to this rapidly spreading killer virus has been exceptional. The spread, the death, the vaccines, all have come in such a short period of time.
The famous company Sherlock Bioscience developed a COVID-19 diagnostic technique based on CRISPR that got approved by the US Food and Drug Administration by the end of May 2020. (4)
By seeing the pace and demand of diagnosing and managing COVID-19, we are sure that CRISPR/Cas9 will soon make its way to the general public (at least for COVID).
Limitations Of CRISPR
There are certain limitations that have restricted the use of CRISPR to clinical trials only. The limitations include:
CRISPR/Cas9 treatments are not 100 efficient. In some cases, there has been no genome editing activity after insertion.
Difficulty To Deliver
Delivering the CRISPR/Cas9 system to mature cells in a living human being remains a problem. Viral vectors are the most common form of delivery method, however, their safety is not sure.
Despite it being accurate, the technique is not 100% accurate and at times there are unwanted “off-target” edits that can lead to severe consequences.
There exists a global moratorium which means a ban on changing heritable DNA to make genetically modified children. This moratorium doesn’t necessarily mean a complete ban, but only restricts germline editing using any technique. (5)
This issue, however, remains at the state level. These limitations shall subside once the demand for treatment outweighs the ethical obstacles.
CRISPR In The Future: A Ray Of Hope
More than 51% of respondents in the US, Canada, France, Belgium, and Australia are willing to consume foods prepared with CRISPR technology. (6)
These results suggest that the public is ready and willing to accept CRISPR in their daily lives. This is crucial because public acceptance determines whether a product will survive in the market or not.
As per research by Yang et al., improvements in editing capabilities due to continuous efforts to understand pitfalls will allow the full potential of CRISPR to benefit the society in near future. (7)
The biggest ray of hope for an early arrival of CRISPR for the general public is the authoritative statements by pharma company heads.
John Leonard, the CEO of Intellia Therapeutics is of the view that CRISPR gene-editing system/treatment can reach patients very, very soon. Owing to the therapeutic benefits imparted by the CRISPR/Cas9 technique, the general public and desperate patients are eager to get a solution to their problems.
As per Leonard, “CRISPR is a major advance in the gene editing space” and will be accessible by everyone pretty soon. The claim comes after successful trials of treating a rare disease with this modern technique of gene editing.
Therefore, all you waiting for CRISPR to go public must be a little more patient as the tool is on its track!
- Zhang, Han, and Nami McCarty. “CRISPR‐Cas9 technology and its application in haematological disorders.” British journal of haematology 175.2 (2016): 208-225.
- Kaiser, Jocelyn. “Tweaking genes with CRISPR or viruses fixes blood disorders.” (2020): 1254-1255.
- Ledford, Heidi. “CRISPR treatment inserted directly into the body for first time.” Nature 579.7798 (2020): 185-186.
- Arnold, Carrie. “What’s new in clinical CRISPR?.” (2021): 184-185.
- Lander, Eric S., et al. “Adopt a moratorium on heritable genome editing.” (2019): 165-168.
- Yang, Yue, et al. “CRISPR/Cas: advances, limitations, and applications for precision cancer research.” Frontiers in Medicine 8 (2021).