TechnologyGene Editing and Therapy - 9 Amazing Facts

Gene Editing and Therapy – 9 Amazing Facts


In Layman’s Language, it can be explained to a five-year-old this way. Similar to drawing and colouring beautiful pictures, the bodies of animals are composed of tiny fragments called GENES which are the major factor behind our facial features, body language and intelligence. We inherit these ‘data units’ from our ancestors.

However, sometimes, these genes get mixed and this gives birth to variations and abnormalities.

It is here that Gene Editing and Therapy come into the picture!

Scientists and researchers figured out a way to ‘repair’ these mixed-up genes using a technique of Gene Editing called CRISPR!

They use special tools called CRISPR scissors to snip and edit the genes, correcting them and all again!

Gene Editing and Therapy is like a rebirth- a blessing for people who have genetic abnormalities and ailments. Gene Editing is a big adventure in the land of biology where scientists explore the secrets of life and unlock amazing discoveries!

1 Introduction

In the contemporary world of cutting-edge technologies and scientific advancements, one special and unique field has emerged that has unprecedented scope for transforming the way we interpret and cure genetic diseases- Gene Editing and Therapy.

By utilizing the might of molecular biology and genetic engineering, scientists have unlocked the capability to manipulate the blueprint of life-giving way to a bright future where correction of genetic disorders will not be a privilege. This revolutionary technology, though still in its nascent stages, holds the promise of transforming lives and reshaping the landscape of healthcare.

The concept of manipulation of the genetic code of our bodies forms the heart and soul of Gene Editing and Therapy. DNA, which stands for Deoxyribonucleic Acid, is a sequence of nucleotides that carry information about our body’s functioning and maintenance.

However, sometimes, variations called mutations arise in the genetic composition that can cause serious diseases like Sickle Cell Anaemia.

In the older times, when Technology was not sophisticated enough to deal with such complexities, major emphasis was laid on controlling the symptoms rather than getting rid of the root cause. However, with gene editing and therapy, a new era of precision medicine dawns upon us.

Gene Editing and Therapy
Image by PublicDomainPictures from Pixabay Copyright 2013

2 CRISPR: Tool behind Gene Editing and Therapy

CRISPR CAS-9, a creative first-of-its-kind gene Editing technology has transformed the area involving genetic research and therapy. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It is a natural system, originally discovered in bacteria that acted as a defense system against viruses. Scientists utilized this system for humans and converted it into a successful and powerful tool for gene Editing.

The CRISPR-Cas9 system consists of two essential components: the Cas9 protein and a guide RNA molecule. The guide RNA molecule directs the Cas9 protein to the desired location within the genome, where it cuts the DNA strand. This cut triggers the cell’s natural DNA repair mechanisms, which can be exploited to introduce specific genetic modifications. Researchers can either insert new genetic material, remove existing segments, or modify specific nucleotides, thus allowing them to edit genes with accuracy and precision.

This CRISPR-Cas9 has a vast range of implications for fields, ranging from basic research to human health. Basic research, enables scientists to study gene function in detail and disclose the sophisticated mechanisms at play underlying different diseases and ailments. In agriculture, CRISPR helps to increase yields of crops and improve the nutritional content, while simultaneously making them resistant to damage by pests.

Gene Editing and Therapy
Image by Arek Socha from Pixabay Copyright 2017

3 Impact On Human Health: Gene Editing and Therapy

One of the most promising applications of CRISPR lies in human health. It offers a revolutionary breakthrough in curing genetic disorders by correcting the genetic mutations right from their source.

Hence, these hold immense scope for the treatment of sickle cell anaemia, cystic fibrosis and some types of cancer. This technology could transform the personalized medicine domain by altering an individual’s genetic makeup.

So, CRISPR Cas-9 has emerged as a revolutionary tool for gene Editing and Therapy, with a wide range of implications.

Image by  PublicDomainPictures from Pixabay Copyright 2013

4 Gene Editing Techniques and Tools

Gene Editing and Therapy have transformed biotechnology the hold a gigantic scope for curing genetic ailments and disorders. The tools and techniques behind Gene Editing have changed drastically over the years, supplying great precision and efficiency in terms of alteration of genetic makeup.

One of the most commonly used tools in gene Editing is the CRISPR system. CRISPR and its sibling protein, Cas-9 work as biological scissors, cutting down the ‘abnormal’ genetic unit by targeting them and replacing or modifying them. This technique allows scientists to edit the DNA sequence with remarkable accuracy by introducing desired changes or correcting mutations.

Apart from CRISPR Cas-9, there is one more mighty tool called ZFNs. ZFNs stand for Zinc Finger Nucleases, these are proteins that are genetically engineered to attach themselves to specific DNA sites. The components of ZFNs enable them to recognize and break DNA at certain locations. By inducing a double-stranded break in the DNA, ZFNs can stimulate the cell’s natural DNA repair mechanisms, leading to gene modifications.

These powerful tools and techniques have given way to important advancements in gene Editing. The future is even more promising, because of the current research going on in the field of Genetic Engineering.

Image by mcmurryjulie from Pixabay Copyright 2017

5 Advantages of Gene Editing and Therapy

One of the most significant and revolutionary merits of Gene Editing Technology lies in its capability to address the source or root cause of the disorder. Instead of merely hiding or treating the symptoms, these technologies tackle the underlying abnormalities, giving way to long-lasting effects. This acts as a ray of hope for the affected individuals and families.

Furthermore, gene editing and therapy have the potential to revolutionize the treatment of previously untreatable diseases. Many genetic disorders currently lack effective treatments or cures.

However, with gene editing techniques, scientists can potentially correct or replace malfunctioning genes, restoring normal cellular function. This breakthrough could transform the lives of individuals affected by conditions that were previously considered incurable, offering newfound hope and improved quality of life.

Moreover, the risk of adverse effects and improvement in safety in medical interventions is assured. By safely targeting specific genes, these techniques can reduce the risk of unintended consequences. Thus, the approach to the treatment of genetic diseases becomes more focused and controlled.

So, Gene Editing and Therapy have a plethora of advantages in the field of healthcare. They have the potential to address the root causes of genetic ailments and offer personalized treatment of ailments that were previously declared untreatable.

Image by Pete Linforth from Pixabay Copyright 2019

6 Disadvantages of Gene Editing and Therapy

One considerable demerit of Gene Editing and Therapy is the fact that unintended locations on the genetic sequence can also become an off-site target. Despite advancements in precision, there is a risk that due to errors or mistakes, sometimes unintended changes can be made to other parts of the genetic sequence. This can be dangerous and can alleviate the genetic disorder instead of correcting it.

The alteration of one unit of a gene may impact the functioning of other units of the same gene, this is called an off-target effect.

The other disadvantage arises due to natural moral and ethical concerns. An individual’s DNA makeup is a creation of God and makes him or her unique. Manipulating the human genome sequence has been titled as ‘playing with God’.

Moreover, it is an expensive procedure, and not all sections of society can afford to get this medical procedure done.

Hence, it highlights the social inequalities, where an individual with the same genetic disorder as another individual, can get his ‘natural blueprint’ corrected because of being wealthy, though both of them were initially sailing in the same boat. This can widen the gap between the privileged and the marginalized.

So, while gene editing and therapy hold immense promise for the future of medicine, it is crucial to acknowledge and address their disadvantages.

Image by Cezary from Pixabay Copyright 2017

7 Future Directions of Gene Editing and Therapy

The future of Gene Editing is extremely promising because technology is paving way for newer applications every passing day. For instance, a technology called CRISPR-Cas 12, has been an exciting discovery.

Cas 9 has been in wide utilisation for gene Editing, but Cas 12 offers more advantages. It has a smaller size and thus can be easily delivered to the targeted location. Hence, the risk of off-target effects is highly minimized. The precision and convenience of gene Editing are thereby multiplied, paving the way for new avenues for therapeutic interventions.

Another area of exploration is base editing, a novel approach that allows for the precise modification of individual DNA bases, without introducing double-stranded breaks.

As opposed to traditional gene editing techniques that rely on introducing foreign DNA sequences, base editing enables the direct conversion of one DNA base to another. This technology has the potential to correct point mutations associated with genetic disorders, such as sickle cell disease or cystic fibrosis, by editing the DNA at the specific location of the mutation. Base editing is thus a more targeted and safer approach for gene correction.


Image by mcmurryjulie from Pixabay Copyright 2017

8 Way Ahead 

In a world where the boundary between science and imagination once appeared to be thick, techniques like Gene Editing have opened the gateway to a new frontier in medicine. These revolutionary technologies hold the scope to reshape the essence of the survival of the human race.

With the power of cutting-edge technology, scientists have learned to manipulate the intricate tapestry of our genetic code for the betterment of humanity.

Imagine a world where inherited diseases no longer exist, and not a soul is born with any genetic abnormalities. Gene Editing is like a master painter that beautifully and skillfully chisels away at flawed genes.

With each cut, the rays of hope of a brighter future can be seen emerging. Through the marvels of technologies like CRISPR-Cas9, scientists have been able to recompose the symphony of life itself DNA. The melody of potential treatments and cures resonates with a harmonious promise of the future.

9. Conclusion 

The spread of the realm of diseases can be cut, and endless opportunities can be opened. Gene editing transcends the boundaries of the physical, reaching into the realm of human imagination.

Gene editing has already demonstrated its effectiveness in various applications, from correcting single-gene disorders like sickle cell anaemia and cystic fibrosis to tackling complex diseases such as cancer and HIV. These advancements highlight the scope of gene Editing in mitigating the impact of genetic diseases and significantly improving patient outcomes.

Gene editing and therapy represent a transformative frontier in medicine. With their ability to precisely alter genes and potentially cure genetic diseases, these technologies hold immense scope for the future of medicine and healthcare.

By navigating the ethical considerations and ensuring equal access to people from all walks of life, we can harness the power of gene editing to alleviate human suffering, extend lives, and prosper in a brand-new era of personalized medicine.

The journey towards fully realising the potential of gene editing and therapy may look too complex at first, but the potential benefits make it a path worth pursuing the betterment of humanity.

The masterpiece of gene editing and therapy unfolds not only in laboratories and clinics but also in the hearts and minds of individuals. We must cultivate a society that fosters inclusivity and equal access, where brushes of opportunity and knowledge are freely bestowed upon all.

By cutting down barriers and embracing collaboration, we create a vibrant mixture of possibilities, where every stroke of innovation finds its place in the tapestry of humanity.

As we unlock the secrets of our genetic blueprint, we open the gate to an era of commendable transformation. From the discovery of the structure of DNA to changing an individual’s entire genetic composition, we as a race have come far.

We glimpse a world where individuals can not only correct genetic deficiencies but also unlock hidden talents and potentials. The realm of human potential stretches beyond the horizon, beckoning us to redefine the entire definition of being a human.


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