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Genome Editing and Its Potential - An Overview

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Genome editing, also known as gene editing, is a development in biological tools with a broad range of possible applications.

Written by

Hemamalini. R

Medically reviewed by

Dr. Kaushal Bhavsar

Published At July 13, 2023
Reviewed AtJuly 13, 2023

Introduction:

Genome editing techniques allow researchers to alter DNA, changing physical characteristics like eye color and disease risk. Scientists use a variety of tools to do this. These technologies cut the DNA at a precise location. The DNA can be removed, added, or replaced where it was cut. The first genome editing methods were created in the late 1900s. Recently, a new genome editing technology called CRISPR, developed in 2009 has made modifying DNA simpler than ever. CRISPR is easier, quicker, less expensive, and more precise than earlier genome editing techniques. CRISPR is currently widely used by scientists who conduct genome editing. Let's talk about genome editing in greater detail in this article.

What Is Genome Editing or Gene Editing?

Genome editing, also known as gene editing, is a development in biological tools with a broad range of possible applications. Similar to text editing on a computer, genome editing is simple to use. First, a precise search inside the organism's genome ("text") is performed to discover the location where a specific alteration in the DNA sequence ("letters") is needed. The genome editing tool then functions as a set of biological "scissors" to cut DNA in between the "letters" to "label" that location. The final step is a natural process that uses the cell's repair system, which can add or remove "letters" or "edit" them, to achieve the required DNA sequence ("letter") modifications (substitute one letter for another). These modifications to an organism's own "letters" (DNA) produce a particular trait.

What Is CRISPR?

CRISPR (clustered regularly interspaced short palindromic repeats) is also known as CRISPR-Cas. This abbreviation refers to the CRISPR/Cas system, which allows researchers to alter genes. It is a recently created, effective, and adaptable technique for genome editing. Cas protein and guide RNA make up this invention. The Cas protein is guided to a specific area of the genome by the RNA. After that, the Cas protein will cut the DNA. Cells will start a repair process once the DNA has been damaged, which may result in DNA alterations.

What Is the Difference Between Gene Editing and GMOs?

GMOs (genetically modified organisms) are primarily used to describe an organism that contains unusual genomic patterns. This newly inserted DNA may have come from a sexually suitable or unrelated creature. Researchers have even created GMOs using DNA from the same creature but in different configurations. GMOs can also be found in bacteria, fungi, and other creatures, even though the term is most frequently used to refer to plants. Gene editing enables scientists to make extremely focused alterations to an organism's genetic code. Sometimes, these alterations can have a positive impact on agriculture, the environment, or our understanding of biology. Most gene edits alter existing DNA rather than adding new genetic material. Recent advances in gene editing have made it possible to precisely insert desired genetic material into predetermined regions of an organism's DNA.

What Are Genome Editing Methods?

The genome editing technique demonstrates amazing progress in this sector while also emphasizing the importance of basic scientific research in the development of research tools and future disease remedies. Some genome editing methods are given below-

  • Homologous Recombination - Homologous recombination was the first technique researchers utilized to alter the genomes of living cells. The exchange of genetic material between two comparable (homologous) strands of DNA is known as homologous recombination.

  • Zinc-Finger Nucleases (ZFN) - Researchers began utilizing zinc-finger nucleases (ZFN) in the 1990s to increase the precision of genome editing and decrease off-target modifications. ZFNs were created by modifying naturally occurring proteins that were found in eukaryotic species. These proteins can be modified by scientists to cut DNA and bind to particular DNA sequences in the genome. The ZFNs cut the genome at the designated location after binding to their target DNA sequence, enabling researchers to either delete the target DNA sequence or replace it with a new DNA sequence through homologous recombination.

  • Transcription Activator-Like Effector Nucleases (TALENs) - Transcription Activator-Like Effector Nucleases (TALENs), a new class of proteins, were introduced to the field of genome editing in 2009. They are made from naturally existing proteins and have the ability to bind to specific DNA sequences.

Does Gene Editing Affect the Environment or Biodiversity?

Gene-edited organisms have the potential to have a beneficial, negative, or neutral impact on the environment and biodiversity. Gene editing is a technique for altering a specific region of an organism's genome. The genetic component that was altered ultimately will determine the effects of these modifications on the environment, not the gene-editing procedure itself. There are numerous instances when gene editing has been used to safeguard biodiversity and the environment. Moreover, gene editing can provide previously unknown ways to enhance environmental stewardship.

Why Do Scientists Use Gene Editing in Agriculture?

Gene editing is an advanced breeding method that allows for rapid and precise changes to the genomes of agricultural, livestock, and microbial species crucial in agriculture. The tool of gene editing enables scientists to modify an organism's existing genetic coding. These adjustments can occasionally result in significant gains, such as boosting drought resistance, raising yields, or lowering methane emissions. Both traditional approaches like standard breeding and gene editing enable researchers to address new demands in agriculture.

What Can Genome Editing Bring to Healthcare?

In medicine, Advanced Therapy Medicinal Products (ATMPs), the vast majority of which are NGT-products, have enormous potential to treat currently incurable genetic disorders, and rare conditions, and provide patients with long-lasting and life-changing treatments. Some therapies target the underlying cause of the condition, giving patients hope of a cure with a single intervention. Sometimes, cell and gene therapies are created precisely for a single patient, resulting in personalized medicine. ATMPs are being produced or utilized to treat a variety of rare hereditary disorders, including Crohn's disease, epilepsy, Parkinson's disease, Alzheimer's, spinal muscular atrophy, rheumatoid arthritis, diabetes, and rare blood and skin cancer.

Conclusion:

The development of genome editing has the potential to alter how to produce food, detect, prevent, and treat diseases, as well as how to generate energy, and optimize industrial processing. This novel science enables scientists to investigate, modify, build, and recreate extraordinarily complicated pathways, DNA sequences, genes, and natural biological systems. Genome editing tools are able to change cell fate and behavior for the next generation of advances in synthetic biology and gene therapy. Hence, we can learn more about the most difficult biological questions in existence and find solutions that will benefit both the human situation and the environment.

Frequently Asked Questions

1.

Is Genes Editing Before Birth Possible?

Yes, gene editing before birth is technically possible through a technique known as germline gene editing. It includes genetic changes to gametes precursor cells, eggs, sperm, or early-stage embryos in a laboratory.

2.

How Risky is Gene Editing?

Yes, gene editing can be risky; gene editing can lead to technical and serious health risks. Potential risks include off-target effects (unwanted alterations or edits in unintended locations), mosaicism (where not all cells carry the intended genetic changes), and long-term health effects, such as toxicity, inflammation, and increased cancer risk.

3.

Is it Healthy to Edit Gene?

The health outcomes of gene editing depend on the purpose of the edits made. Gene editing holds the potential to treat currently incurable genetic disorders, and rare diseases and provide individuals with long-lasting and life-changing treatments. However, editing for non-therapeutic reasons or enhancing traits raises ethical concerns and potential health risks.

4.

How Successful is Gene Editing?

Gene editing has succeeded in various applications, including research, agriculture, and potential treatments for genetic disorders. However, the success and safety of gene editing in humans are still areas of active research and development.

5.

Is Natural Alteration of Genetics Possible?

Genetic alterations can occur naturally through processes like mutation, often caused by environmental and lifestyle factors. However, directly changing the DNA sequence through natural means is not possible. It can be typically achieved through a gene-editing system known as CRISPR-Cas9.

6.

Is Customization of Baby’s Genes Possible?

Customizing baby genes involves manipulating the DNA of embryos to introduce desired traits or eliminate disease-causing mutations. This is typically done through in vitro fertilization (IVF) combined with gene-editing techniques like CRISPR-Cas9.

7.

How is Pre-Birth Gene Editing Beneficial?

Gene editing before birth, or germline editing, holds immense promise for preventing and treating devastating genetic diseases. This approach is particularly valuable for addressing diseases that prove fatal during pregnancy or perinatal stages or for conditions where irreversible disease processes begin before or shortly after birth. Thus improving the lifespan and overall health.

8.

What Technology Is Used for Gene Editing?

CRISPR Cas9 is a widely used technology for gene editing. Other methods include homologous Recombination, Zinc-Finger Nucleases (ZFN), and Transcription Activator-Like Effector Nucleases (TALENs).

9.

Is it Safe to Editing Genes in Embryos?

The safety of gene editing in embryos is a subject of ongoing research and ethical debate. Concerns include off-target effects, unintended consequences, and the potential for long-term health risks. Rigorous testing and ethical guidelines are essential to ensure the safety of such procedures.

10.

Are Genetically Modified Foods Safe?

The safety of gene-edited foods depends on the specific modifications made. Regulatory bodies assess these foods' safety based on the introduced traits and potential allergens. The process of gene editing itself does not necessarily make foods unsafe.

11.

How Is Genetic Technology Beneficial?

- Genetic technology has numerous benefits, including advancements in medicine, agriculture, and environmental conservation. It can lead to


- Better disease diagnosis and treatment.


- Improved crop yields.


- Development of genetically modified organisms with enhanced traits.

12.

How Promising is Gene Therapy?

Gene therapy holds great promise for treating genetic disorders and certain diseases. Ongoing research aims to improve the efficiency and safety of gene therapy techniques, making them more widely applicable in the future.

13.

How Successful Has Gene Editing Been?

Gene editing has been successful in various contexts, such as modifying cells in the laboratory for research purposes and treating certain genetic disorders in animals. However, challenges and uncertainties remain, especially when applying gene editing to humans.

14.

How Safe Is Gene Editing for Humans?

The safety of gene editing in humans is still under investigation. While progress has been made, potential health risks, technical risks, and ethical considerations highlight the need for cautious and well-regulated approaches to ensure the safety of individuals undergoing gene-editing procedures.

Dr. Kaushal Bhavsar
Dr. Kaushal Bhavsar

Pulmonology (Asthma Doctors)

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