Wednesday, April 13, 2016

CRISPR/Cas9 Research Paper

Kendal Gast
3-22-16
ENGL 355

CRISPR/Cas and the Crispy Road Ahead

            The human race has reached a tipping point.  Or one could argue that we humans have reached several tipping points (realizations) and already forgotten them, only to continue and perhaps, remember, again.  The ability to edit the human genome is hailed by some as the next step in human evolution or a gross overestimation of our hasty, naïve ambition.  Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is a bacterial defense mechanism that has been altered in a way to effectuate genetic engineering (eugenics).  This technology should only be utilized to cure genetic disease, but not used to enhance humans or modify diseases.  CRISPR calls into question what it means to be human, and in the future will need a necessary redefinition.
            CRISPR was first identified as a bacterial acquired immunity function.  Scientists noticed repeated segments of DNA, the palindromic portion of the acronym, in bacteria.  These repeated segments were separated by different, unique patterns.  These unique patterns (protospacers) were observed to be copies of viral DNA the bacteria encountered (Addgene).  A short distance away from these repeats, genes that code for proteins called “CRISPR associated proteins” or Cas were found (Addgene).  The process of defending against known viruses begins by transcribing part of the CRISPR system (or several protospacers), into which becomes pre-CRISPR RNA (Addgene).  The pre-crRNA is then separated into individual crRNAs by trans-activating crRNA (tracrRNA), which in turn brings along RNase III (a ribonuclease) and Cas enzymes to help separate the pre-crRNA (Addgene). The tracrRNA and Cas enzymes combine with the individual crRNA strands to form unique complexes (Addgene).  These complexes then look for complimentary strands of the crRNA, or known sequences of viral DNA.  In order for the complexes to bind to the viral DNA, Protospacer Adjacent Motifs (PAM) must be after the crRNA site (Addgene).  Once the crRNA is matched, the Cas nuclease snips the DNA in both places after the PAM. (Addgene).  Put a little more simply, a large section of the CRISPR system in the bacterial DNA is transcribed.  Similar protospacers (not a part of the original DNA) work with a ribonuclease and Cas enzyme to break up the large section of this CRISPR system into smaller segments.  These segments then bind with the similar protospacers and Cas enzyme to identify the portion of viral DNA to break it, rendering it unusable.  This process occurs naturally in prokaryotic cells, while the cleverness of human scientists (eukaryotic organisms), in modifying this process, but has been adapted to work in mammalian cells - and even fertilized eggs. 
            Scientists can edit DNA sequences in eukaryotic cells by combining crRNA and tracrRNA into a single guide RNA as Rath et al. point out in Biochimie (125).  This allows for a double strand break in DNA when the new guide RNA and target DNA match up (Rath et al. 125).  There are then two ways in which the DNA strand can be repaired:  Non-Homologous End Joining (NHEJ) or Homology Directed Repair (HDR).  NHEJ simply joins the two ends together with a few pairs or deletes other bases to continue the pattern (Rath et al. 125).  This can result in poor functioning of the cell or render the DNA strand obsolete.  HDR, on the other hand, uses another strand of DNA that matches up with both broken ends of the strand (Rath et al. 125).  CRISPR is also able to facilitate multiple strand breaks at once, thus giving scientists the ability to insert several different pieces of DNA or removing large portions of the sequence altogether.  Although this method is much more efficient and precise, compared to just four years ago, it is nevertheless limited by the need of specific PAM sites to direct the Cas enzymes (Rath et al. 125).  CRISPR has fundamentally changed the way scientists conduct everyday eugenic research, yet accrediting the proper individual or group for inventing the technology has proven difficult. 
            The Broad Institute and University of California Berkeley are the two main institutions trying to patent the CRISPR/Cas system.  As Tony Fong writes, “In April [2014], the US Patent and Trademark Office issued the first patent, No. 8,697,359, for the CRISPR-Cas9 system to the Broad Institute” (Genomeweb).  Because of this, Feng Zhang of the Broad Institute founded Editas Medicine in November 2013 with UC Berkeley scientist Jennifer Doudna, among others (Fong).  Having cooperating scientists claim that they each invented the technology makes it harder to award patents, difficult to utilize the technology, and also not make any sense.  On top of all this, Doudna gave a TED talk at the end of last year and explicitly stated that she was the inventor of the CRISPR technology.  Things get even more confusing because Doudna and Emmanuelle Charpentier, professor of Hannover Medical School in Germany, co-authored a paper in August 2012 after working together and sharing data on Cas enzymes (Fong).  Then, of course, Charpentier told The Independent that she came across the technology first (Fong).  A number of other institutions have filed patents parallel to the CRISPR system before any of these notable scientists came on to the scene (Fong).  The Broad Institute’s application could be limited by an approval of UC Berkeley’s application, which will have effects in how CRISPR develops and is distributed for wider use after the USPTO decides what to do.  Garden-variety scientists know more about how this system functions in such a short amount of time and somehow immediately see the profitable applications a technology this powerful has to offer.  Because of CRISPR’s far-reaching and relatively unknown future, Doudna and other think tanks have voiced their concerns about human applications, especially anything to do with human embryos.
            Perhaps what is most disturbing about a gene editing technology is the uncertainty it brings.  Human beings have only recently discovered the ability to manipulate DNA directly, and luckily it has been too difficult to change human DNA.  Gretchen Vogel writes that in 1975, scientists, policymakers, and lawyers gathered at Asilomar, California to “craft[ed] guidelines for research that altered the DNA of living organisms” (1301).  Back then, the idea of altering human DNA seemed too extraordinary for the current moment then.  In January of this year, Doudna organized a similar meeting, albeit smaller, of experts to discuss guidelines for the safe and ethical use of CRISPR methods (1301).  Two group commentaries published in Science and Nature both asked for serious consideration of action before any scientist attempts to modify human DNA for clinical uses (1301).  In the Nature commentary, the authors asked for a complete prohibition “on any experiments that involve editing genes in human embryos or cells that could give rise to sperm or eggs” (1301).  Doudna and co. on the other Science hand, do not insist on an all-stop measure, but instead “strongly discourage” any attempts at human DNA modification for “clinical applications” – with the exception of some human cells allowed as long as no one tries to get pregnant (1301).  In fact, there are no laws in the United States or China that ban germline genetic engineering (1301).  However, if one wishes to establish a pregnancy in the U.S. using this method, it needs approval from the FDA (1301).  Think tanks like the Hinxton Group have already proposed guidelines for this type of research.  Last September they released their “Consensus Statement on Genome Editing Technologies and Human Germline Genetic Modification” (hinxtongroup.org).  It describes very clearly 12 issues that scientists and governments need to address if they are to continue moving forward with DNA modification technologies, which, as the authors are already aware, will happen (hinxtongroup.org). 
            But these journal articles and grand statements of issues are too shortsighted.  They miss the point of a few years longer down humanity’s road, probably about 20 years.  Several of the citations used in this paper mentioned how easy it would be for an individual with amateur biology, genetic, and lab knowledge to utilize CRISPR and modify nearly any cell or prokaryotic organism they desire.  That is the world of 2016.  The world of 2036 will most likely have altered human beings at a fundamental level, and diseases as well.  Sure, scientists would have already cured most genetic diseases by then, but why should they stop there?  At some point the human race will have crossed the threshold of God-power.  No longer will there be a clear distinction between “humankind/man” and “nature”.  There very likely will only be “humankind/man”.  A valid argument can be made that this is the obvious next step for humans.  No longer can they plod along, wondering if they will ever evolve into something different, better, greater.  Humans will take it upon themselves to make sure their species embodies “progress” in the absolute, most magnificent sense of the word.  Like the narrator says of Jimmy in Oryx and Crake, “Perhaps he failed to take seriously his own despair” (344).  As do humans. 



Works Cited
Addgene. Addgene.org. Oct. 2015. Web. 1 Mar 2016.
Atwood, Margaret. Oryx and Crake. New York: Anchor Books, 2004. Print.
Fong, Tony. “As CRISPR-Cas9 Technology Sets to Take Off, Uncertainty Swirls Around
            IP Landscape.” Genomeweb. Ed. Ed Winnick. 18 Jun 2014. Web. 9 Mar 2016.
Hinxton Group, The. Hinxtongroup.org. Johns Hopkins Berman Institute of Bioethics.
            3 Sept 2015. Web. 8 Mar 2016.
Rath, D. Amlinger, L. Ruth, A. Lundgren, M. “The CRISPR-Cas Immune System:
            Biology, Mechanisms, and Applications.” Biochimie 117: Special (2015): 119-
            128. Web. 8 Mar 2016.
Vogel, Gretchen. “Embryo Engineering Alarm.” Science 347.6228 (2015): 1301. Web.
            8 Mar 2016.

            

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