Gene Editing – Unlocking the Potential Within

The editing of genes alters a living cell’s genetic material (DNA or RNA). In order to add, delete, or replace individual genetic bases and sequences, it utilizes a variety of different methods and techniques. In medicine, the gene editing process has facilitated the study of diseases in detail, helping clinicians and researchers to understand their root causes. 

The most significant aspect of gene editing is this emphasis on causes as well as on treatment. Although gene editing has been used mainly as a medical biotechnology, it also has exciting applications in many other areas, including agriculture and biofuels, where it can produce more disease-resistant strains of crops or algae. 

As it impacts the building blocks of life, gene editing is a controversial technology, sometimes raising public concerns. However, its growing usage cannot be overlooked and, if potential negative impacts are to be handled, knowledge of its applications is important. Enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, are used to edit genes, where they introduce cuts into the DNA strands, allowing existing DNA to be extracted and replacement DNA added. 

To do this, scientists use various technologies. Such techniques behave like scissors, cutting the DNA at a particular location. Then the DNA where it was cut can be extracted, inserted, or substituted by scientists. In the late 1900s, the first technologies for genome editing were established. More recently, DNA editing has been made simpler than ever by a modern genome editing technique named CRISPR, invented in 2009.

Correcting Genetic Mistakes to Invention of Gene Therapy:

In the genetic discovery period of the mid-20th century, researchers discovered that the sequence of bases in DNA is transmitted from parent to offspring. Recognition of the latter led to the inescapable conjecture that the means to fix those errors would come with the discovery of “molecular errors” that cause genetic diseases and thus allow disease prevention or reversal. 

The underlying concept behind gene therapy was the notion which was used in molecular genetics as a holy grail from the 1980s. However, it has proven difficult to establish gene editing technology for gene therapy. Many early developments focused not on resolving genetic errors in the DNA, but rather on trying to minimize their effect by supplying a functional copy of the mutated gene, either incorporated into the genome or retained as an additional chromosomal unit (outside the genome).

Process:

Genome editing is a procedure where the genetic code of an organism is modified. Researchers use enzymes to ‘cut’ DNA to create a double-strand break (DSB). Non-homologous end joining (NHEJ) or homology-directed repair occurs via DSB repair (HDR). NHEJ creates random gene knockout mutations, while HDR uses extra DNA to construct a desired sequence within the genome (gene knock-in). 

There are four Gene Editing Techniques: Tools to Change the Genome:

Sr.No.	Techniques	Principle
1	Restriction Enzymes: the native Gene editor	In the 1970s, with the discovery of restriction enzymes, the ability to edit genes became a reality. Restriction enzymes identify and cut unique nucleotide sequence patterns at that site, providing a chance to inject new DNA material at that location.
2	Zinc Finger Nucleases (ZFNs): Increasing  identification	ZFNs consist of two parts: an engineered nuclease (Fokl) fused to the DNA-binding domains of the zinc finger. A 3-base pair site on DNA is identified by the zinc-finger DNA-binding domain and can be merged to identify longer sequences.
3	TALENs Gene Editing: Potentiality within single nucleotide	Transcription activator-like nucleases of effectors (TALENs) are similar to ZFNs structurally. Both methods use the Fokl nuclease to cut DNA and involve functioning dimerization, but the DNA binding domains vary. TALENs, tandem arrays of 33-35 amino acid repeats, use transcription activator-like effectors (TALEs).
4	CRISPR-Cas9 Gene Editing: The game changer	CRISPR consists of a guide RNA and a Cas9 nuclease and is a two-component system. Within the ~20 nucleotide region identified by the guide RNA, the Cas9 nuclease cuts the DNA. Knocking out particular genes in cell lines to interrogate gene activity is one of CRISPR’s most commonly used applications.

 

Promoting the Sustainable Development Goals (SDGs)

Gene editing has the ability for many of the SDGs to be advanced. Some examples of areas of application across a wide range of sectors are given below. 

SD Goals	Applications
2. Zero hunger	Develop the ability of crops to thrive in areas constrained by capital.  Manage in a humane and ethical way the stock and productivity of livestock.
3. Good health and wellbeing	Instead of treating symptoms, which is the current emphasis of most medical medicine, cure or stop diseases. Within the larger trend towards providing genomic medicine, studying the genetic make-up of a person will determine whether a patient will respond well to a drug treatment and allow targeted treatments that reduce unpleasant or harmful side effects.
6. Clean Water and Sanitation	Dissimal and removal by gene editing tools and systems biology of the persistent xenobiotic portion from water have emerged as the outstanding alternative. To overcome the difficulties in the field of bio-remediation of recalcitrant substances from the environment, several bioremediation approaches are present.
7. Affordable and clean energy	Develop new sources of energy by allowing organisms to generate biofuels more effectively, such as bio ethanol. This would help minimize reliance on energy sources that are not renewable or detrimental to the environment, such as fossil fuels.
13. Climate action	To enhance the use of photosynthesis, gene editing plants may become even more effective in trapping and sequestering carbon from the air.

 

Conclusion:

Advances in genome editing methods have opened new doors to what genome editing can do to solve medicine, agriculture, and beyond problems. CRISPR has fully revolutionized what genome editing, by growing the pace and scope of research, will mean for our future. In its role in drug development, diagnostics, and gene drives, we are already feeling the impact of CRISPR, just to name a few. At this pace, don’t be shocked if in the near future you see more discussion about genome editing.