The world of Gene editing— Think about it!
What does biotechnology behold for the future? Nanotechnology, stem cells, phage therapy? We are living in a time where we can literally impact billions, with science!
What is Gene editing?
Such biotechnology, a major player in biology, is Genome editing. Genome editing technologies enable scientists to make changes to DNA, changes in physical traits, like eye colour, and disease risk. These technologies act like scissors, cutting the DNA at a specific spot. Then scientists can remove, add, or replace the DNA where it is cut.
How Gene editing works
Genome editing uses a type of enzyme called an ‘engineered nuclease’ which cuts the genome in a specific place. Engineered nucleases are made up of two parts:
- A nuclease part that cuts the DNA.
- A DNA-targeting part is designed to guide the nuclease to a specific sequence of DNA.
After cutting the DNA in a specific place, the cell will naturally repair the cut. We can manipulate this repair process to make changes (or ‘edits’) to the DNA in that location in the genome.
CRISPR
CRISPR “spacer” sequences are written down into short RNA sequences (“CRISPR RNAs” or “crRNAs”) capable of guiding the system to matching sequences of DNA. When the target DNA is found, Cas9 — one of the enzymes produced by the CRISPR system, binds to the DNA and cuts it, shutting the targeted (tiny chemical assembly instruction inside of living things) off. Using changed versions of Cas9, (people who work to find information) can activate (tiny chemical assembly instruction inside of living things) expression instead of cutting the DNA. These ways of doing things allow (people who work to find information) to study the gene’s function.
Research also hints that CRISPR-Cas9 can be used to target and change “typos” in the three-billion-letter sequence of the human (total set of tiny chemical assembly instructions of a living thing) to treat (related to tiny chemical assembly instructions inside of living things) disease.
How does CRISPR-Cas9 compare to other genome editing tools?
CRISPR-Cas9 is proving to be an efficient and customizable alternative to other existing genome editing tools. Since the CRISPR-Cas9 system itself is capable of cutting DNA strands, CRISPRs do not need to be paired with separate cleaving enzymes as other tools do. They can also easily be matched with tailor-made “guide” RNA (gRNA) sequences designed to lead them to their DNA targets. Tens of thousands of such gRNA sequences have already been created and are available to the research community. CRISPR-Cas9 can also be used to target multiple genes simultaneously, which is another advantage that sets it apart from other gene-editing tools.
How is Gene editing being used right now?
There are various uses of Gene editing, which is being limited to healthcare. To expand its functionality and develop new applications for genome editing, we must evaluate several examples of its current use.
Healthcare
Cancer research:
Genome enhancing science has done fundamental centred dividing occasions in a variety of essential studies, from its preceding proofs of environment-friendly gene enhancing in eukaryotes to its latest purposes in the engineering of hematopoietic stem cells (HSCs) and tumour-targeted T cells; this science has installed novel standards of gene change and has held to a border subject of most cancers research.
Cardiovascular disease:
CVD is a serious hazard to human health and is the number one cause of death in many industrialized countries. Many different types of CVD are usually associated with a single genetic mutation or a combination of rare inherited heterozygous mutations. In practice, clinical treatments focus on the relief of disease symptoms without addressing potential genetic defects. Currently, the establishment of in vivo CVD models with gene-editing technology and the in-depth analysis of CVD pathogenic genes as well as their molecular mechanisms have made it possible to test the ability of gene therapy to control specific gene expression and improve gene functions. With the help of genome editing technologies, various research models of cardiovascular conditions have been created.
There are many diseases gene editing has been helping to cure!
Gene editing effects
CRISPR genome editing may result in unwanted heritable genetic changes, which could lead to long-term risks in a clinical context. Three independent studies published on the preprint platform bioRxiv have reported unintended DNA changes adjacent to the target site when using CRISPR/Cas9 in human embryos.
Concerns that people have about gene editing are:
- There is a high chance of errors occurring during the gene-editing process. Errors can have devastating consequences. For example, a researcher can accidentally delete a gene. This can lead to developmental defects in the fetus. Any errors in germline editing could be passed on from generation to generation.
- Once people have access to the technology, it might be hard to control what it’s used for. This could create a slippery slope. Parents-to-be might use the technology in ways that are considered sexist or racist. For example, if parents can choose their baby’s sex, is this a way of allowing sexism? If parents can choose physical traits that are more common in races they find more attractive, is this a form of racism?
Companies working with Gene editing
CRISPR Therapeutics:
At CRISPR Therapeutics, they aim to advance transformative gene-based medicines based on CRISPR/Cas9 gene editing. For genetically-defined diseases, we can use a guide RNA that directs Cas9 to cut DNA at a specific site in a disease-causing gene, or at a different site, such as a region that regulates genes, to ameliorate the genetic defect through gene disruption or correction. For cell therapies, we can target genes that when disrupted may improve the safety or ability of the therapy, or precisely insert new genes to give the cells new abilities. In either case, we may edit cells either ex vivo (outside the body) or in vivo (inside the body).
Editas Medicine:
At Editas Medicine, they are exploring the possible. There mission and commitment is to harness the power and potential of CRISPR gene editing to develop a robust pipeline of medicines for people living with serious diseases around the world. There goal is to discover, develop, manufacture, and commercialize transformative, durable genomic medicines for many diseases.
Intellia Therapeutics:
There researchers work tirelessly to harness the genome editing technology CRISPR/Cas9 for human therapeutic use. Jennifer Doudna, an Intellia co-founder, and Emmanuelle Charpentier were awarded the 2020 Nobel Prize in Chemistry for their pioneering work in CRISPR. They at Intellia are humbled to have a hand in making what we believe to be medical history.
They are employing a modular genome editing platform to create diverse in vivo and ex vivo pipelines, spanning a range of therapeutic indications. Guided by this full-spectrum approach, They are committed to making CRISPR/Cas9-based medicines a reality for patients suffering from genetic diseases and to creating novel engineered cell therapies for various cancers and autoimmune diseases.
Impact of Gene-editing in the future
Gene editing, although it has been popularized in the past decade, has the potential to grow. As of right now, we’re just unlocking some of this potential by targetting various problems ranging from paralysis to treating seizures. But that is not all! In fact, some claim that we will be able to use gene editing to communicate with one another just by thinking about it! The possibilities and growth of gene-editing technology are vast, and we are yet to see the next evolution of mankind!
