CRISPR Gene Editing

The CRISPR Gene Editing Approach to Treating and Curing Diseases

The Promise

DNA (Deoxyribonucleic acid) is the “source code” of life, and now we can edit and correct that code efficiently and cost-effectively should there be a disease-causing error. In other words, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)[1] gene editing could save millions of lives and cure thousands of genetic disorders, if it is to fulfill its promise.

Many diseases arise due to “typos” or mutations in our DNA. In fact, since reading the human genome for the first time successfully in 2003,[2] researchers have discovered more than 10,000 diseases that result from single gene mutations.[3] Many of these disorders have no known remedy. Although each mutation affects only a small population, given the sheer number of rare diseases hundreds of millions of people suffer.[4]

CRISPR gene editing could be the tool that enables clinicians to correct these genetic typos, providing these populations with a cure. Moreover, the technology should give researchers a better understanding of how genetic mutations influence complex diseases. In short, the discovery and commercialization of CRISPR finally could herald the age of personalized medicine, making genetic information consistently actionable.

What Is CRISPR?                              

CRISPR refers to a DNA sequence encoded in a bacterial genome that protects it from viral invaders. In the bacterial genome, the DNA sequence latches on to a pre-specified portion of the virus’s DNA and, using a pair of molecular scissors, disables it at that spot. In 2012, scientists realized that CRISPR could be re-engineered and redirected to attach to any stretch of DNA in other organisms.[5] In effect, they found a gene-function disabling tool that would direct them to most targets of interest.

We think CRISPR gene editing is analogous to a DNA word processor with two functions: FIND and DELETE. In addition, scientists are working on a rudimentary PASTE function, allowing CRISPR to insert appropriate DNA code to repair mutations. While CRISPR is analogous to a DNA word processor, previous techniques mimic old-fashioned typewriters, requiring actual physical cutting and pasting.[6]

Looking Into the Future

  1. CRISPR likely will become a common research tool in the discovery of underlying biological mechanisms.
  2. CRISPR potentially will improve early-stage therapeutic research by reducing labor hours and increasing R&D productivity. CRISPR can reduce the time not only to genetically engineer mice by 75% (to just six months) but also to create mutant mice by 30%.[7]
  3. Non-human organisms likely will provide fertile ground for CRISPR to launch. In contrast to the rigor required for human clinical trials, traditional gene splicing techniques have become commonplace in agriculture and biofuel research and development, paving a path for increasingly complex CRISPR applications.
  4. The next few years will be pivotal in demonstrating CRISPR’s utility in human health, particularly rare genetic disorders. Based on ARK’s projections, the first CRISPR-generated cures are due to come to market before 2020.
  5. Currently, CRISPR-based editing still produces a number of off-target cuts, a known risk that scientists are addressing. The FIND-DELETE function may find and delete unintended regions of the genome, a key weakness of the technology. As scientists continue to develop and perfect the DELETE function of CRISPR, the FIND feature already has proven to be useful as a low-cost diagnostic tool for infectious diseases.[8]

The potential for CRISPR gene editing is obvious, and the benefits to human health could be remarkable. We look forward to delving into more details about CRISPR specifically comparing it to other gene editing techniques, in a blog to follow.

 

[1] This blog uses CRISPR to broadly address all CRISPR subtypes. The CRISPR gene editing system is comprised of a guide RNA and an enzyme that is responsible for splicing. Scientists have discovered a number of enzyme variations that work with the CRISPR system. http://www.popsci.com/new-alternative-crispr-enzyme-could-make-genetic-edits-more-precise

[2] https://www.genome.gov/11006943/human-genome-project-completion-frequently-asked-questions/

[3] http://www.who.int/genomics/public/geneticdiseases/en/index2.html

[4]350 million people globally are estimated to suffer from rare diseases, of which 80% are of genetic origin. https://globalgenes.org/rare-diseases-facts-statistics/

[5] There are some limitations as to where it can attach and cut, but this blog will leave those complications aside for now.

[6] Scientists have been able to manipulate the genome previously using bio-engineering and Zinc Finger Nucleases (ZFNs) http://www.nature.com/mt/journal/v23/n5/full/mt201554a.html

[7] Before a therapeutic program enters human trials, it must clear animal safety and proof of concept tests. The complexity of an engineered mouse determines costs; however, one lab reduced costs to engineer one strain of mutant mice to just $100,000. http://www.sciencemag.org/news/2016/11/any-idiot-can-do-it-genome-editor-crispr-could-put-mutant-mice-everyones-reach

[8] Low-cost Zika virus diagnostics using CRISPR-single resolution assays http://www.cell.com/pb-assets/journals/research/cell/cell8923.pdf


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