CRISPR-Cas9 is a powerful gene editing tool that allows scientists to make precise and targeted changes to the DNA of an organism. Biochemically, Cas9 is a naturally occurring 160 Kilodalton protein in bacteria that is used to defend against invading viruses and plasmids.

Magically after its first practical demonstration in 2008 by Eugene Koonin and colleagues at the National Center for Biotechnology Information in Bethesda, Maryland, this tool is extensively used in genetic engineering.

The  sequences by combining the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system with the Cas9 enzyme. The CRISPR system employs  as a guide molecule to target a specific place on the DNA. The Cas9 enzyme subsequently slices the  at the desired spot, allowing particular  sequences to be inserted, deleted, or substituted. The CRISPR-Cas9 system’s precise nature makes it a strong tool for editing genes, and it has the potential to transform medicine by allowing for the repair of genetic defects, the discovery of novel  therapies, and the development of more efficient and sustainable crops.

CRISPR, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats is a DNA sequence found in the genomes of bacteria and archaea. These sequences are transcribed into short RNA sequences called the CRISPR RNAs or crRNAs which are capable of guiding the Cas9 system to matching sequences of DNA.

Cas9 is a nuclease enzyme capable of cutting the DNA at the targeted site.

The guide  molecule is coded with the CRISPR sequence and the target sequence to guide to a specific sequence in the DNA. Upon identifying the site, the Cas9 nuclease enzyme chops off the DNA at that site.

Using the natural DNA repair mechanism in the cell, this cut is shortly paired. It results in the insertion or deletion of specific genes or the correction of mutations.

CRISPR-Cas9 gene editing tool
Image: CRISPR-Cas9 | Creative Commons

Since its development,  sequences by combining the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system with the Cas9 enzyme. The CRISPR system employs  as a guide molecule to target a specific place on the DNA. The Cas9 enzyme subsequently slices the  at the desired spot, allowing particular  sequences to be inserted, deleted, or substituted. The CRISPR-Cas9 system’s precise nature makes it a strong tool for editing genes, and it has the potential to transform medicine by allowing for the repair of genetic defects, the discovery of novel  therapies, and the development of more efficient and sustainable crops.

CRISPR-Cas9 has become one of the most widely used and versatile gene editing tools and has been applied in a variety of fields, including medicine, agriculture, biotechnology, and basic research.

CRISPR gene editing and its applications in medicine

CRISPR gene editing is a revolutionary new tool in molecular biology and virtually has infinite potential. Because of the practicability and preciseness of the  sequences by combining the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system with the Cas9 enzyme. The CRISPR system employs  as a guide molecule to target a specific place on the DNA. The Cas9 enzyme subsequently slices the  at the desired spot, allowing particular  sequences to be inserted, deleted, or substituted. The CRISPR-Cas9 system’s precise nature makes it a strong tool for editing genes, and it has the potential to transform medicine by allowing for the repair of genetic defects, the discovery of novel  therapies, and the development of more efficient and sustainable crops.

CRISPR gene editing is a revolutionary new tool in molecular biology and virtually has infinite potential. Because of the practicability and preciseness of the  sequences by combining the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system with the Cas9 enzyme. The CRISPR system employs  as a guide molecule to target a specific place on the DNA. The Cas9 enzyme subsequently slices the  at the desired spot, allowing particular  sequences to be inserted, deleted, or substituted. The CRISPR-Cas9 system’s precise nature makes it a strong tool for editing genes, and it has the potential to transform medicine by allowing for the repair of genetic defects, the discovery of novel  therapies, and the development of more efficient and sustainable crops.

CRISPR-Cas9 system can be used for an array of applications, like the correction of genetic mutations and diseases, the development of new treatments, and the creation of animal models for studying human diseases.

  • Gene therapy for inherited genetic diseases

CRISPR-based gene therapy is a promising approach for the treatment of inherited genetic diseases. Genetic diseases are caused by mutations in a person’s DNA. Gene therapy targets correcting the underlying genetic defect to cure or alleviate the symptoms of the  once and for all.

It is possible because this tool allows for precise modification of the genome. By delivering the CRISPR machinery (the guide  and the Cas9 nuclease) directly to the affected cells, it is possible to repair the mutated DNA in-vivo to restore the function.

Some examples of inherited genetic diseases that are being explored for CRISPR-Cas9-based gene therapy include:

  • Cystic fibrosis: a lung  caused by mutations in the CFTR gene.
  • Sickle cell anemia: a  disorder caused by mutations in the HBB gene.
  • Huntington’s disease: a neurodegenerative disorder caused by a mutation in the HTT gene.
  • Hemophilia: a bleeding disorder caused by mutations in the F8 or F9 genes.

Correction of genetic mutations that cause cancer and other diseases.

CRISPR
Image:  Council

CRISPR can be used to correct genetic mutations that cause cancer and other diseases.  is often caused by mutations in genes that regulate cell division and growth which leads to uncontrolled cell proliferation and the formation of tumors.

CRISPR is implemented to precisely edits these mutated genes making it possible to restore normal function and potentially cure cancer.

In addition, it can be used to target the gene-specific mutations in a patient’s tumor, allowing for the creation of personalized treatments tailored to each individual’s unique genetic profile. Certain other diseases caused by genetic mutations, like muscular dystrophy and beta-thalassemia, may also be amenable to CRISPR-based gene therapy.

  • In developing new treatments for viral infections.

When specific viral genes are inactivated, the replication and spreading are disrupted, thus effectively

curing the infection. Researchers are now actively exploring the modification of genes of viruses such as human immunodeficiency virus (HIV), hepatitis B and C, and influenza using CRISPR technology.

Yet another approach targets engineering human  to be resistant to viral infections. This is done by editing the genes that the virus normally targets.

  • Creation of personalized medicine through genetic engineering.

Also known as precision medicine, CRISPR has the potential to play a major role in the creation of personalized medicines. Precision medicine is an approach that tailors medical treatment to the individual characteristics of each patient, including their genetics, environment, and lifestyle.

First, CRISPR analyzes a patient’s genetic profile and identifies specific mutations or variations. Then customized gene therapies can be designed to target the underlying causes of their disease. For instance, personalized cancer therapies are being researched that to target specific mutations in a patient’s tumors. Thus, treatments tailored to an individual’s genetic profile can be created.

In a different approach, the gene editing tool can also be used to develop personalized vaccines. This is done by targeting specific mutations in a virus or bacteria unique to a patient under consideration.

  • Generation of animal models

CRISPR
Image: Animal models | Genome.gov

CRISPR technology has revolutionized the field of  by creating animal models. Using those models, the underlying causes of human diseases, development, and testing of new treatments or cures can be analyzed without using an actual human being.

Animal models are created by modifying the genes of animals to make them closely resemble human diseases, including genetic disorders and complex diseases such as cancer and heart disease. Researchers have already created  with this technology that carry specific mutations associated with human diseases, such as Alzheimer’s and Huntington’s.  organism.

The animal models for the study of viral infections like HIV and Influenza also have shown promising results. It was done by engineering animals that are susceptible to these viruses. Studying these animal models sometimes becomes crucial to identify the course of the infection and newly made vaccines.

The dangers advancing with CRISPR-Cas9

Although CRISPR-based gene therapy is a promising field, it raises some crucial ethical and safety concerns. The dangers are alarming as its open validation and use upon living creatures can do more harm to the world than benefit.

Numerous examples have raised these safety concerns. There is barely any method to monitor whether the CRISPR machinery delivered to the  of the patient’s body has not caused any unintended consequences. Hence, despite the promises, this technology must be validated with technical hurdles before it can be used directly by human beings.

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