In the last few years, CRISPR technology has captured the attention of the scientific community and the rest of the world. It is one of the most important scientific breakthroughs after mapping the human genome. Although most of the CRISPR-based platforms are still at nascent stages, CRISPR is already beginning to reconstruct the physical world around us in less radical ways, one base pair at a time.
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What is CRISPR? Why is there so much hype around it?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeat. The name refers to the type of repeated DNA sequences found in the genome of bacteria and microorganisms. CRISPR was discovered after biologists observed that some bacteria were able to cut the DNA as a self-defense mechanism.
Historically, gene editing was an expensive, labor-intensive process that required biologists to load genes onto viruses bound for target cells. However, today biologists can easily and quickly edit DNA as computer engineers change codes. CRISPR is the easiest, fastest and cheapest technology used by biologists/scientists to modify the genetic code. Application of CRISPR within healthcare has ranged from drug discovery to the possibility of xenotransplantation.
So how does CRISPR work?
CRISPR technology requires three elements to work
- Cas9 enzyme,
- RNA sequence, and
- a DNA template
Biologists start with RNA. It is responsible to locate the spot in the cell nucleus where the edit needs to occur. The Cas9 enzyme is linked to the RNA guide, which goes through the sequence of DNA letters in the genome looking for a match. When the matching target sequence is located, the Cas9 enzyme splices the target DNA. This triggers the cell to try and repair the sequence with any DNA that is available. Hence, we inject the DNA material alongside the RNA sequence and Cas9 enzyme so that the cell can use the new DNA sequence to repair the cut. However, in absence of additional DNA material, the cell will repair by joining the cut ends.
6 Healthcare Verticals Gene-editing tool CRISPR Could Disrupt
Here are the six healthcare verticals that could fundamentally change with CRISPR application
1. Cancer Therapeutics
Scientists are experimenting with CRISPR to discover new immunotherapies to treat cancer. CRISPR has enabled gene editing for precision cancer modeling and lets scientists better understand the effects of cancer and ways to treat it. Scientists could genetically engineer T-cells (immune cells) using CRISPR to find and kill cancer cells. Cancer patients can be injected with these modified T-cells to fight off cancer.
2. Genetic Diseases
Researchers speculate that CRISPR could potentially treat genetic diseases such as diabetes, muscular dystrophy, cystic fibrosis, genetic blindness and more. Genetic disease is caused by a mutation in DNA sequences that are responsible to perform a particular function. The scientific community has already identified many genes that are crucial in causing genetic disease. Therefore, CRISPR could be used to repair faulty genes and possibly cure genetic disorders.
CRISPR Therapeutics is focused on developing breakthrough medicines using its proprietary CRISPR/Cas9 gene-editing platform to treat patients with serious diseases.
Fertility is another area where CRISPR could have a profound influence. 15 to 20 percent of pregnancies in the US end in a miscarriage and identifying genetic causes underlying miscarriages is extremely challenging. Researchers have used CRISPR to understand infertility so that they could find treatments.
Researchers at The Francis Crick Institute used CRISPR to stop a gene from producing a crucial protein called OCT4 that causes embryos to collapse. This is the first time that a study of this nature has been conducted in human embryos. CRISPR application is still at early stages in this industry and the concept of designer babies remains contentious.
Current diagnostic methods require patients to visit clinics to extract a biological sample to send to a lab for diagnostics. This process takes a significant amount of time. Scientists have unveiled a CRISPR protein that targets RNA (instead of DNA) to be used as a rapid and inexpensive tool for disease diagnosis. The tool can be designed as a paper-based test which will not require refrigeration and therefore, can be used outside of traditional lab settings.
Mammoth Biosciences is developing an at-home CRISPR diagnostics kit which aims to provide clinical grade diagnosis from a paper strip. All one needs will be 30 minutes and a smartphone.
5. Organ Transplantation
In the United States, a person is added to the national transplant list every 10 minutes and 20 people die each day waiting for a transplant (Network for Organ Sharing). Demand for human organs far exceeds the supply. Fortunately, scientists are predicting that CRISPR could help change this situation.
Pig organs are similar in size and function to humans; however, pig’s genome consists of 25 retroviruses that might transmit diseases to people. Using CRISPR–Cas9 gene-editing system, scientists were able to remove all 25 viruses in the pig genome, resulting in healthy piglets and pushing research closer to xenotransplantation – a process that involves the transplantation of either live cells, fluids or organs from animals to human recipients.
Start-up eGenesis, founded by CRISPR pioneers, applies gene-editing tools to find the possibilities of xenotransplantation.
6. Drug research and development
Scientists are speculating that CRISPR technology could potentially speed up the drug discovery process which generally tends to be long, expensive and regulated. By applying CRISPR, researchers can identify the genes that cause or prevent diseases and develop treatments accordingly.
In addition, CRISPR makes it easier to create cellular and whole organism models that precisely mimic diseases. This helps scientists to better understand the efficacy of drugs, providing better predictions in clinical trials.