Redefining Possibilities: Eight diseases CRISPR technology could cure

CRISPR/Cas9 was first utilized as a gene-editing theorem in 2012. In just a few years, the technology has exploded in popularity thanks to its promise of making gene editing faster, cheaper and easier than ever before.

Cancer

Cancer is a cure remaining elusive, complex, and multifactorial disease. There are thousands of various types of cancer, each with the mutation signature. CRISPR technology is a gaming-changer for cancer treatment and research as it can be utilized for numerous things, including screen for cancer drivers, identifying genes and proteins that could be targeted by cancer diagnostics, cancer drugs, and as a treatment.

Blood disorders

The blood disorders sickle cell disease and beta-thalassemia, which affect oxygen transportation in the blood, are the targets of a CRISPR treatment being generated by its partner Vertex Pharmaceuticals and CRISPR Therapeutics. 

Blindness

Many hereditary forms of blindness are caused by a particular genetic mutation, making it easier to use CRISPR/Cas9 to treat it by target and modify a single gene. In addition, the immune system activity is limited in the eye, which prevents any issues connected to the body rejecting the treatment.   

AIDS

There are some pathways CRISPR could help us in righteousness against AIDS. One is utilizing CRISPR to cut out the viral DNA that the HIV virus inserting DNA of immune cells within. This approach could be utilized to attack the virus in its hidden, inactive form, which is what makes it impossible for most therapies to completely getting rid of virus.

Cystic fibrosis

Cystic fibrosis is a genetic disease that causes respiratory issues. Although there are treatments accessible to deal with the indication, the life expectancy is only around 40 years. Cystic fibrosis do be caused by various different mutations in the targeting gene CFTR – over 700 of which have been identified – make it tough to develop a drug for every mutation. With CRISPR technology, mutations that cause cystic fibrosis could be individually edited.

Muscular dystrophy

Duchenne’s muscular dystrophy is caused by mutations in the DMD gene, which is an encoding for a protein essential for the muscles contraction. Children born with the disease suffering progressive muscle degeneration, and existing treatments are restricted to a fraction of patients with the condition.  

Research in mice has shown CRISPR technology can be utilized to fix up the numerous genetic mutations behind the disease. In 2018, a group of researchers in the U.S. utilized CRISPR to cut at 12 strategic mutation hotspots covering the majority of the estimated 3,000 various mutations that cause this muscular disease. A company named Exonics Therapeutics was spun out to further develop the approach. A year later, it was acquired by Vertex Pharmaceuticals for approx $1 billion to accelerate disorder drug development.

Huntington’s disease

Huntington’s disease is a neurodegenerative state with a good genetic component. The disease is caused by an unusual repetition of some DNA sequence within the huntingtin gene. The higher the number of copies, the earlier the disease would manifest itself.

Treating Huntington’s can be tricky, as any off-target effects of CRISPR in the mind could have very threatening consequences. To lessen the risk, scientists are looking at pathways to tweak the genome-editing tool to make it safe.

 COVID-19

In the face of COVID pandemic, CRISPR has quickly been put to use for fast screen tests. In the longer term, the gene-editing tool might permit us to fight COVID-19 and other viral infections. 

Another study group at the Georgia Institute of Technology has utilized the same approach to destroying the virus before it enters the cell. The method was tested in live animals, improving the hamsters’ symptoms infected with COVID. The treatment is also working on influenza mice infected, and the study believes it can be effective against 99 percent of all the existing influenza strains.

The future of CRISPR technology

Considering that CRISPR/Cas9 is a newest development in the biology world, study has only begun to scratch the surface of the role it could play in the future. The examples listed here are just the foremost attempts at utilizing CRISPR technology as a therapist. As they progress out, we do expect more and more indicators to be added to the list. 

One of the biggest challenges to turn this research into real cures is the many unknowns regarding the CRISPR therapy potential risks. Some studies are concerned about possible immune reactions and off-target effects as well to the gene-editing tool. But as study progresses, scientists are testing and proposing a huge range of approaches to improve and tweak CRISPR in order to increase its safety and efficacy. 

Hopes are higher that CRISPR/Cas9 technology would soon serve a pathway to target and destroy complex diseases like AIDS and cancer, and even target genes connected with mental illnesses.