Holly Ward

CRISPR is an effective gene editing tool that can alter the DNA of every living thing. Even if CRISPR is able to treat some rare diseases there are ethical concerns. Learn more.

CRISPR is a gene editing tool. It is utilized by scientists to alter or "edit" DNA within living organisms. They've conducted a number of research so far, but they're still years away from using it as part of an approved treatment for individuals.

While scientists are convinced that this tool could bring an important change in the way we manage (or cure) many diseases--including rare genetic disorders and cancers, it's yet too early to know how well it's going to be effective over the long-term.

Researchers believe that this technology could change the way medicine is done and could be able of curing many diseases--including rare genetic diseases and cancers in the future--but it's still too early to determine how effective it is.

Where did CRISPR come from?

Researchers today employ a variety of methods that people studying bacteria had discovered some time ago. CRISPR is just one of them.

CRISPR, short for CRISPR-Cas9, is a special immune system that's in bacteria. It is used to fight off viruses that try to attack them.

This is how it works It's when viruses inject their virus DNA into bacteria to attack them. That's when bacteria start their fight. They use their CRISPR-Cas9 to chop pieces of viral DNA so they're no more an issue.

CRISPR/Cas9 gets its name from two important components of the system:

CRISPR stores DNA fragments that viruses leave behind following an attack. It's like scrapbooks for viruses, and can help bacteria find invaders it hasn't previously encountered.

Cas9 is an enzyme that acts like tiny scissors. It cuts DNA that isn't present.

How does CRISPR work?

Your DNA is a set of genetic instructions you have acquired from your parents. It explains how to make all the various parts of your body. DNA is composed of four chemical bases, or "letters": A (adenine), T (thymine) C (cytosine), and G (guanine). These letters can be combined in a variety of ways to make "words" or "genes".

If parents are passing their DNA to their children, some of the letters may be missing, altered or copied incorrectly. Most of the time, this doesn't make any difference, but time it can affect how a gene operates. This is known as a genetic mutation (variation), and could cause serious issues, such as rare diseases.

When researchers first discovered the CRISPR/Cas9 mechanism in bacteria They wondered if they could use it to help people. They wanted to explore the possibility of eliminating harmful genetic mutations, and replace them with the correct DNA. This might lead to an effective treatment or even cure for various illnesses.

Scientists made modifications to CRISPR-cas9 to enable them to use it in their lab. The CRISPR system can detect defective DNA patterns inside cells of living things. After their CRISPR finds the part of deoxyribonucleic acid that's a problem the Cas9 (the "scissor" enzyme) eliminates it.

If DNA is damaged as this in nature, it heals itself. With CRISPR, scientists can utilize the same process of repair to create new DNA. What is the procedure? They inject new DNA (or genetic genes) into the cell. The new DNA is added to the cell as the DNA repairs itself.

CRISPR may be a significant factor for people with rare diseases as around 5600 (80%) of the rare illnesses have a genetic basis. CRISPR is a great option, however research is still in its early stages. This means it will take many years for this type of gene editing to be approved by the FDA for use in patients.

What are the main issues with CRISPR?

CRISPR's most significant flaw is its inefficiency. It hasn't been able to function in every cell that scientists have examined. Some cells fail to even make it through the process.

CRISPR isn't without ethical and safety concerns.

A few studies suggest that CRISPR might cut out areas of DNA that aren't intended to, which may create dangerous mutations in the DNA of certain cells. This could lead to more health problems than it is trying to fix. This is known as off-target effects.

CRISPR is primarily about creating "designer babies" or making use of Cas-9 to make a statement for aesthetic purposes. Moral concerns with CRISPR are mostly focused on the creation of "better" babies. Some examples include creating a child with a "better" physical appearance or athletic ability, various hair colors, eye colour, and so on. CRISPR is still many steps to go before such genetic alternatives become feasible. To address these fears, the National Academies of Sciences, Engineering, and Medicine have put together a comprehensive report for 2020. It has guidelines and suggestions for using gene-editing tools like CRISPR.

The conclusion

We'll probably hear a lot more about CRISPR in time to come, as scientists make advances as they learn about the advantages and disadvantages of this procedure. But along with the promise of curing rare diseases comes the obligation to use CRISPR in a safe and ethically.