The concept of gene editing may sound futuristic to some. However, gene editing, short for genome editing, has been around since the late 20th century. This technology has allowed scientists for over one hundred years to edit DNA.
More recently, gene editing technology has become more advanced, allowing scientists a faster and more accurate way to edit DNA. This particularly recent breakthrough technology is called CRISPR.
What is CRISPR used for exactly? In this article, we’ll explore how CRISPR works and what this invention has done for gene editing as a whole.
How CRISPR Works
Scientists today are able to quickly and efficiently target and cut DNA through the molecular tool CRISPR. This gene editing technology is short for Clustered Regularly Interspaced Short Palindromic Repeats. Also known as CRISPR-Cas9. These two key molecules will be what introduce change into DNA.
Think of CRISPR as a hand holding a pair of scissors. The hand is a guide RNA meant to find a specific sequence of DNA. Cas9 is the pair of scissors that is there to make a cut at a certain section of two DNA strands. This allows specific sequences to be added or removed at the cut-off section of DNA.
Once Cas9 has snipped a spot of DNA, cells sense a problem and attempt to repair the break. Scientists can then introduce changes to the cell in question. What then is this CRISPR-Cas9 mechanism used for?
Research is of course still ongoing but has been advancing rapidly in recent years. CRISPR is being used in trials to treat various disorders and diseases in both humans and animals.
For example, certain cancers like multiple myeloma, sarcoma, or diseases like sickle cell anemia have seen promising results with Cas9 CRISPR utilization. Hereditary conditions or genetic disorders such as Huntington’s disease are also prime targets for CRISPR therapy.
Who Invented CRISPR?
While genome editing has been around for a while, programmable gene editing at the level that CRISPR brings was invented by Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier.
The convenient and efficient way in which genetic engineering is now possible landed Dr. Doudna and Dr. Charpentier the Nobel Prize in 2020. This recognition came nearly 13 years after the function of CRISPR was first elucidated.
As the technology is being researched and ever-evolving, one particular factor has been shown to help the efficacy of CRISPR’s gene editing abilities. Enter mRNA. A piece of the puzzle that can help encode CRISPR’s abilities.
Kenneth Chien, Co-Founder of Moderna believes mRNA has high potential to be a key component in helping treat certain conditions. This is in tandem with the idea that these sort of technologies can be repurposed to treat diseases of the heart, the nervous system, and beyond.
A Modified Future
After answering the question of how CRISPR works, we can then look to how making these gene changes can help us better understand genetic diseases. In time, and with further advancements in the field, CRISPR will surely change the way we treat human diseases.
The possibilities of CRISPR-based technology are endless. It’s only a matter of time before CRISPR becomes a household name.
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