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Gene-Editing Techniques Could Treat Mutation Diseases

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A group of scientists at Salk Institute for Biological Studies, San Diego have created a tool that can perform gene edits. They say they can do these gene-editing techniques with diseases that are created by Mutation like Huntington’s.

 

The gene-editing techniques, called SATI, build on a variant of the CRISPR-Cas9 gene-editing technique called HITI (homology-independent targeted integration). SATI means ‘Single Homology Arm donor mediated Intro-Targeting Integration’. The technique deals with mutations by inserting a healthy copy of a troubled gene in the non-coding region of the DNA.

 

The DNA then starts to repair itself as the normal gene, and the old one integrates into the genome. This, in turn, eliminates the harm from the mutation disease with no risks. The head of the research team, Juan Carlos Izpisua Belmonte said:

“This study showed that SATI is a powerful tool for genome editing. It could prove instrumental in developing effective strategies for target-gene replacement of many different types of mutations, and opens the door for using genome-editing tools to possibly cure a broad range of genetic diseases.”

 

The SATI Technique

Researchers carried out a lab test to confirm this technique. The test showed that the technique had very significant effects on a premature ageing disease, progeria. Apparently, the mice they treated with SATI lived 45% longer. They also had a significant level of healing from the ailment as the ageing effects also reduced. This would then mean over ten years of extended lifespan for humans affected by the same progeria condition.

 

The Researchers (L-R) Juan Carlos Izpisua Belmonte, Reyna Hernandez-Benitez, Pradeep Reddy and Mako Yamamoto. Photo credit: Salk Institute

 

This is possibly a major science and tech breakthrough. However, there is still so much work to do. The team of scientists look to make the technique more efficient. They specifically aim to boost the number of cells that can incorporate healthier DNA.

 

The lab experiments are also meant to only be a proof of concept. It will take a lot more experiments before scientists can carry out clinical trials. Meanwhile, the success of this technique could eventually help doctors eliminate a wide range of mutation gene diseases.

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