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CRISPETa - A Novel Tool to Delete Disease Causing Genes

CRISPETa - A Novel Tool to Delete Disease Causing Genes

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  • A research team led by Carlos Pulido and colleagues has developed a computational tool which can produce optimized sgRNAs
  • These RNAs can act as molecular scissors that delete specific regions of the DNA
  • When the edited section of the DNA codes for RNA, the RNA also contains the edited versions of the gene sequence.

Genomics and the structure of our DNA have been merely "read" so far; however, scientists have now developed a computational tool that will allow easy deletion of DNA within living cells. The study was published in PLOS Computational Biology and this vital step towards understanding DNA could help scientists gain a better understanding of the non-coding regions of the DNA. The scientists hope that it will aid in identifying certain unknown regions of the DNA which lead to diseases and will result in a potential new method of therapy.

The Dark Matter

The CRISPR-Cas9 editing tool has revolutionized gene therapy by allowing silencing of genes that code for proteins. However, the vast majority of our genes are termed "dark matter" as they do not code for any protein; thus, studying these non-coding genes would help gain key insights into human biology and disease development.



Based on CRISPR-Cas9 gene editing tool, The Johnson lab developed a new tool called "DECKO" that deletes non-coding DNA. This system utilizes 2 sgRNAs that act as molecular scissors and cut out DNA at both ends, which is a unique plus point of this system. Though this tool was accepted by many, there was no software which could develop the two sgRNAs; this made the deletion experiment laborious and lengthy.


Dr. Carlos Pulido and colleagues developed a software called CRISPETa that can perform the deletion experiments using CRISPR. The region of the DNA that needs to be deleted should be entered into CRISPET, and the tool then designs sgRNAs which are then used by the scientists to carry out the experiment. The designs can be generated at high scales, with an eye on future experiments.

Advantages of CRISPETa
  • Optimized pairs of the sgRNA can be designed by the CRISPETa tool
  • The targeted DNA can be efficiently deleted using the CRISPETa designs
  • The system efficiently deletes targets within human cells
  • The RNA that is coded from the edited/deleted sections of the DNA also contains the same editions/deletions
  • The tool has been designed to be used even by people who are not experts, which will allow the tool to be used across various laboratories
  • It could soon be used as a therapeutic strategy, deleting sections of DNA that are found to lead to the development of disease
Dr. Rory Johnson, Head of the Laboratory, said that the CRISPR deletion tools as well as other genome editing tools have revolutionized our ability to understand the human genome. Dr. Johnson believes that it aids in understanding disease development, especially in the 99% of the DNA that do not code for proteins. Though this gene editing tool is utilized currently as a basic research tool, it can be used as a therapeutic tool to reverse mutations that lead to disease.

Dr. Carlos Pulido who wrote the software for CRISPETa said that the new software tool opens a large number of possibilities to utilize CRISPR deletion in their research.

Bringing the Woolly Mammoth Back to Life Using CRISPR Technology

The CRISPR/Cas9 system has been gaining considerable importance in the field of genomic studies. In another interesting study that utilizes the CRISPR/Cas9 system, Dr. George Church recently stated attempting to bring the woolly mammoth back to life. This project is not a vanity project, according to Dr. Church, who has edited 15 copies of genes, out of the 30 that has to be edited in total, to be able to carry out the task.

Dr. Church, from The Harvard Woolly Mammoth Revival Team, believes that repopulating the tundra and the boreal forests of Eurasia and North America with Asian elephants, would help break down big ice deposits thus allowing cold air to pass through. Since the Asian elephants are unable to survive in the cold weather conditions, the scientists are using CRISPR technology to integrate genes from the mammoth genome with genes of the Asian elephants to help them adapt. This is one of the most eagerly awaited projects, with the entire world watching to see if the woolly mammoth will indeed be brought back to life.

The need to bring back the woolly mammoth would aid in understanding the complex evolutionary mechanisms that were necessary to protect species from extinction. It would also serve as a method for large mammal conservation. The Asian elephants are not adapted to survive in cold regions and are thus restricted to living in the Asian regions. However, poaching has led to a drastic reduction in their numbers. Using the CRISPR/Cas9 system to help the Asian elephants survive in the colder climates assists in
  • Adaptation for better circulation of blood and oxygen in the low temperatures
  • Increased subcutaneous fat which will help insulate against cold
  • Growth of additional hair which will protect the elephant from the cold air
Such conclusive steps in genomics have been made possible with the advent of these gene editing tools. There are many diseases that are associated with the junk DNA and, for which there are no current methods of therapy. The CRISPETa is a potential therapeutic strategy which could delete sections of DNA that are found to result in the development of disease.

References :
  1. Woolly Mammoth Revival - (http://reviverestore.org/projects/woolly-mammoth/)
Source: Medindia

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