Genome (Mojica et al., 2005). That finding took

Genome
Editing

Genome editing
is a hot topic in science world today. Particularly, the CRISPR-Cas9 system
that proved to be faster, cheaper and efficient genome editing tool. The tool
enables altering genes permanently at precise locations. The implications may
include prevention of wide varieties of diseases like cancer and mental
illnesses in the future. Various ethical issues preceded the emergence of the
CRISPR system around the world.

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Scientists
alter an organism’s DNA using genome editing. The recent tool for gene editing
incorporates CRISPR: Clustered Regularly Interspaced Short Palindromic Repeats and
Cas9 enzyme. CRISPR was characterized in 1993 by Francisco Mojica from
University of Alicante, Spain. In 2005, he reported that these repetitive sequences
matched snippets from the genomes of bacteriophage (Mojica et al., 2005). That
finding took him to a correct hypothesis that CRISPR is an adaptive immune
system. (2)

How
does it work? CRISPR sequences are transcribed into a piece of guide RNA that
was pre-designed. It is located within a longer RNA and leads Cas9 to the aimed
part of a gene. Cas9 enzyme cuts two strands of a double helix DNA at a
targeted location in order to add or remove DNA at that place. The molecular
cut is made that way. Later, the cell will try to repair the “damage”. Thus, cell’s
natural occurring DNA repair systems are used in this technology. (3)

The
importance of gene editing encompasses prevention of numerous medical
conditions. Research is still on the way to determine if it is safe to use the
method on humans. Feng
Zhang, was the first to successfully adapt
CRISPR-Cas9 for genome editing in eukaryotic cells (Cong et al., 2013). He
has also published numerous papers like “Multiplex Genome Engineering Using
CRISPR/Cas Systems”, “RNA targeting with CRISPR–Cas13”, “RNA-guided human
genome engineering via Cas9” and more. The CRISPR Genome Engineering Resources
website is linked to his lab and provides vast variety of information on CRISPR
tools, resources, troubleshooting tips and more. The website also provides link
to an open to public, free, annual Genome Engineering 5.0 workshop that was
held on May 7-9, 2017 at Broad Institute in Cambridge, Massachusetts. (5)

CRISPR-Cas9
system is carried on somatic, non-reproductive cells. Nevertheless, a
controversy arose due to a proposal of using germline cells, which are egg and
sperm cells. Because the gene editing made on those cells will be passed on to
the next generation. (3) Ethical issues like that include embryo genome
editing, which is banned in many countries in the world. Yet, scientists from
China publicized their embryo editing work in 2015. Experiments used nonviable
embryos and resulted in unplanned edits in their genes apart from the targeted
gene. Another Chinese group of researchers from Guangzhou Medical University
tried to repair disease mutations in viable embryos. But the edited embryos
possessed gene mosaicism in their genome. In addition, on July 26th
the MIT Technology Review announced that genome editing experiments have
happened in the United States as well. Although not published, it is known that
the research is led by Shoukhrat Mitalipov of Oregon Health and Science
University in Portland. Tens of effectively edited embryos were produced
without mosaicism in their genome, which suggests that by far it is the most
successful attempt. (6)

In
conclusion, genome editing has been a controversial issue in the world of
science. CRISPR-Cas9 gene editing system proved to be fast, efficient and
cheaper than the preceding systems. It is also predicted to be used in treating
many medical conditions in humans in the future. Annual genome editing workshops
hosted in Broad Institute prove that genome editing has a bright future. And
even though sex cells and embryo editing is prohibited in many countries,
Chinese and the U.S. scientists has recently announced their progress in that
field. 

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