1. What is CRISPR?

CRISPR (pronounced “crisper”) is a term often used as shorthand to refer to the ‘CRISPR-Cas’ gene editing technology that, since 2013, has revolutionised the biotech world.

 

There are two elements to a CRISPR-Cas system:

  • The scissors for cutting DNA (this is the Cas bit); and

  • The mechanism to guide the scissors to the bit of DNA that you want to cut (this is the CRISPR bit). 

 

In fact, there are at least 16 different CRISPR-Cas systems recognised today.  The most common one being used in labs around the world is CRISPR-Cas9.

Accordingly, CRISPR is essentially a tool for editing DNA.

      

DNA is the molecule carrying genetic instructions used in all known living organisms and scientists have been tinkering with and manipulation DNA for many years.

The key shift is that CRISPR now enables genes to be edited with a much greater level of precision and flexibility.  This has generated much excitement throughout the scientific community.

 

2. What has it achieved to date?

There has been a flurry of recent scientific CRISPR related activity including the notable achievements listed below, all of which are “firsts” in their fields.  The superscripts that map to the related web-links provided in the footnotes below.

 

  • The prevention of HIV infection in human cells.  See footnote 1.

  • The creation of monkeys with pre-specified, targeted genetic mutations. See footnote 2.

  • The engineering of the first ever “semi synthetic” organisms.  See footnote 3.

  • The first ever successful treatment of a genetic disease in an animal.  See footnote 4.

3. What could it achieve in the future?

The key points here are:

  • This treatment is not solely targeting embryos but also living humans.  Many animal studies have already been carried out.

  • The “cutting technology” is undergoing continuous refinement aimed at greater accuracy, thereby also helping to eliminate any unintended consequences. See footnote 5.

  • Clinical trials involving humans are now underway in China.  To be more specific, individuals with lung cancer have been subjected to injections with cells that have been modified using the CRISPR gene-editing technique. See footnote 6.  

  • The first human trials in the US are expected to start in early 2017.  This will target three genes in participants’ cells with the goal of treating various cancers.  In March 2017, a group at Peking University in Beijing hopes to start three clinical trials using CRISPR against bladder, prostate and renal-cell cancers.  See footnote 6.

According to Carl June, the scientific adviser to the planned US trial, this could trigger “… a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product”.

It is the outcome of such trials that form the basis of our CRISPR based longevity catalyst.  The precise formulation is as follows:

"Clinical trials carried out on humans yield validated outcomes that result in FDA or EMA approval for a CRISPR based treatments (primarily targeting various forms of cancer) being fast tracked for large scale development."

4. How does it work?

There are many sources on the web that describe this in varying levels of detail and indeed clarity. 

 

The Working Party’s view of the most helpful such sources are listed below:

http://www.yourgenome.org/facts/what-is-crispr-cas9

 

http://sitn.hms.harvard.edu/flash/2014/crispr-a-game-changing-genetic-engineering-technique/

 

https://en.wikipedia.org/wiki/CRISPR

 

https://www.broadinstitute.org/what-broad/areas-focus/project-spotlight/questions-and-answers-about-crispr

 

https://motherboard.vice.com/en_us/article/what-is-crisprcas9-and-why-is-it-suddenly-everywhere

 

https://www.youtube.com/watch?v=MnYppmstxIs

 

http://gizmodo.com/everything-you-need-to-know-about-crispr-the-new-tool-1702114381

References

  1. http://www.pnas.org/content/111/31/11461.abstract 

  2. http://www.nature.com/news/first-monkeys-with-customized-mutations-born-1.14611

  3. http://www.sciencealert.com/new-organisms-have-been-formed-using-the-first-ever-6-letter-genetic-code

  4. http://www.sciencealert.com/crispr-gene-editing-tool-used-to-treat-genetic-disease-in-an-animal-for-the-first-time

  5. https://www.statnews.com/2016/04/20/clever-crispr-advance-unveiled/

  6. http://www.nature.com/news/crispr-gene-editing-tested-in-a-person-for-the-first-time-1.20988

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