Crispr might help cure your genetic disease with a gene-editing technology, CRISPR

Crispr

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Crisper technology has far-reaching consequences in terms of human gene editing, and experts are confident about treating human genetic diseases with gene editing technology. CRISPR-Cas9 first came into the picture as a gene-editing tool in 2012. Within just years, this technology has exploded in rage due to its future aspects of making gene editing much easier, cheaper, and faster.

 

What is CRISPR?

It stands for ‘clustered regularly interspaced short palindrome repeats.’ This term basically refers to a combination of repetitive patterns found in the DNA of bacteria. With the help of CRISPR, we can cut off bacteria’s DNA to treat them. It works by replacing the defected DNA by simply providing it with an RNA template of the target sequence.

 

Effects of CRISPR gene editing

It has already revolutionized the way scientists used to perform research, with extensive applications across multiple fields. In the future, CRISPR has the potential to let us edit any genetic mutation for the cure of any disease with a genetic origin. However, CRISPR is still in the early stages of its therapeutic development.

Here’s the list of genetic disorders that scientists hope to cure with CRISPR technology.

Blindness

Scientists have found that it’s possible to cure blindness with the help of CRISPR-Cas9. According to researchers, a specific type of hereditary blindness that is caused by a specific genetic mutation may be treated by targeting and modifying a single gene. 

Furthermore, a company, Editas Medicine, has undertaken treating inherited child blindness with CRISPR therapy for Leber congenital amaurosis. The cure focuses on repairing the function of light-sensitive cells before the children completely lose sight.

The experiments conducted last year are expected to give results by 2024. It’s a new technology in which genes editing occurs directly inside a patient’s body.

 

Covid-19

During the Covid-19 pandemic, CRISPR has been in service to increase the screening tests. In the longer term, scientists think that the way CRISPR has proven beneficial against Covid-19, prospects are very high that we could use it against other viral infections.

At Stanford University, scientists have developed a technique to create the gene-editing technology known as Cas13a that may be able to eliminate the genetic material of the virus. This approach has demonstrated a positive outcomes in minimizing the infectious load in human cells by 90%.

Similar results were obtained at the Georgia Institute of Technology when this approach was applied to the virus before it enters the cell. However, the test was performed on hamsters infected with Covid-19, which demonstrated signs of improvements.

 

Cancer

China is the pioneer in testing the first clinical trials using CRISPR-Cas9 as a cancer cure. The clinical trials were aimed at modifying the T cells extracted from the cancer patient. The scientists tested this method on 12 patients at West China Hospital. Moreover, the outcomes suggested that the gene-editing technology was feasible and safe.

In this trial, scientists removed the PD-1 gene that is responsible for protein-encoding because this protein is usually targeted during cancer treatment, such as checkpoint inhibitors.

In the US, a similar approach is reviewed by the University of Pennsylvania, and the researchers were able to remove three genes that help cancer evade the immune system.

 

Blood disorders

Fatal diseases that cause thousands of death every year, beta-thalassemia and sickle cell anemia, are on the radar of CRISPR treatment. A cure developed by CRISPR Therapeutics and its partner Vertex pharmaceutical is expected to treat these diseases using gene-editing technology.

This treatment is possible by extracting the patient’s bone marrow from stem cells, and the CRISPR would be used to produce fetal hemoglobin. With this method, oxygen-carrying protein could bind oxygen in much better ways, treating cells to be reinfused into the patient.

Scientists hope to cure other blood disorders that have remained untreated until now, such as Hemophilia.

 

AIDS

AIDS has long remained one of the fatal diseases that killed thousands of people, but now CRISPR could help us in the fight against AIDS. One approach is to edit the Viral DNA, which has been inserted inside the patient by HIV, with the gene-editing method. This method might be able to identify and kill the inactive and hidden form of the virus, which makes it difficult for most therapies to completely get rid of the virus.

Another technique could be used to make us strong against HIV infections. Some people are born with biological resistance to HIV, which is due to CCR5 that encodes a protein on immune cells. The mutation in protein can make the virus ineffective.

 

Huntington’s disease

This disease is an outcome of a neurodegenerative condition in which an abnormal repetition of a DNA sequence happens within the huntingtin gene. As the copies number of DNA rises, the chances of manifesting disease itself also increase.

It’s a challenging task to treat Huntington’s as any side effects of gene-editing technology in the brain could have very fatal consequences. To minimize the risk, scientists are scrutinizing several ways to adjust the CRISPR in the brain. In 2018, researchers demonstrated a new version of CRISPR that contains a self-destruct button. For more precise gene editing, Polish scientists relied on pairing CRISPR-Cas9 with an enzyme called nickase.

 

Cystic fibrosis

Cystic fibrosis is a genetic disorder that inflicts several respiratory problems. Though it’s a curable disease with current technologies, the life experience of patients is only around 40 years. Scientists are working on this disease to find its origin so that the gene could be cured, which causes cystic fibrosis.

Last year, an approach was adopted which could use bad editing to repair genes without causing much damage elsewhere in their genetic code. Moreover, several pharma companies have plans to introduce proper treatment for cystic fibrosis using the gene-editing method.