Molecular Biology DNA Notes

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In the 1980s, molecular biologist Dr. Yoshizumi Ishino and his team noticed an unusual
repetitive DNA sequence in an E.coli genome which, unbeknownst at the time, was the first
encounter with Clustered regularly interspaced short palindromic repeats (abbreviated CRISPR)
that has revolutionized molecular biology in recent years. The function of CRISPR and CRISPR
Associated (abbreviated Cas) system was part of prokaryotic immune systems in which a Cas
complex is able to protect the cell from plasmids and phages by cleaving a region of DNA. As a
result, scientists were able to develop a genome editing tool known as the CRISPR/CRISPR
Associated Protein 9 (abbreviated Cas9) system. A study in 2012 by Doudna, Charpentier, and
Martin Jinek showed that the guide RNA could be engineered, expanding the possibilities of
genome editing technology.
Here’s how it works: The system is able to cleave DNA at a specific sequence with a protein
known as the Cas9 endonuclease. First, the Cas9 endonuclease matches its guide RNA to the
corresponding sequence in the DNA via base pairing. Then, the Cas9 protein is able to cut out
the specific sequence of DNA by cleaving the phosphodiester bond between two
deoxyribonucleotides. The resulting gene is thus altered.
With the discovery of CRISPR, many insist the genome editing system could play a huge role in
global development. In Bill gates’ article in Foreign Affairs on Gene Editing for Good, CRISPR
could potentially be used to assist in improving life expectancy, fighting poverty, or promoting
food security for developing nations in many aspects.
Applications of CRISPR/Cas-9 systems can be used for human germline gene editing and
“normal…pre-implantation development” research. The information from this type of research
could be useful with potential solutions and treatments for infertility and other reproductive
issues.
The CRISPR/Cas9 system is still a relatively new discovery, so despite its potential to address
issues such as poverty and illness, there are many risks and concerns around its application.
Ethical concerns related to CRISPR mostly surround human germline gene editing (abbreviated
GGE) because the edits made would be passed down to future generations.
The application of GGE comes with two potential implications: edits in the wrong place, known
as off-target effects, and mosaicism: the chance that not all cells carry edited genes. This poses
a concern on the safety of this technology, suggesting that the risks greatly outweigh the
potential benefits. The concept of informed consent for germline gene therapy (CRISPR) is also
being discussed as the affected patients are embryos, or rather, future generations. Moreover,
even with the presence of the parents, informed consent is hard to obtain as long as the risk of
this technology is unknown. Thus, bioethicists and researchers insist on further research before
the use of (GGE) for reproductive purposes is attempted. Additionally, like many new
technological advancements, there is a concern that access to CRISPR/Cas-9 genome editing
will only be a privilege of wealthy individuals, resulting in increased existing disparities in access
to healthcare. Finally, ethical debates also surround varying morals and religious beliefs of
individuals as the technology is being used on human embryos.
In 2018, discussion of regulations and laws surrounding CRISPR genome editing in human
embryos became heavily debated in the scientific community. A Chinese scientist named He
Jiankui used the CRISPR/Cas9 system to edit the genomes of two embryos. The embryos were
then implanted and born several months later, being the first CRISPR babies. The scientist
aimed to use CRISPR/Cas9 genome editing to reduce the risks of getting human
immunodeficiency virus, HIV, by deleting a DNA sequence thought to be associated with the
virus’ ability to infect cells. Dr. He Jianku faced incredible backlash from the scientific community
after presenting his research at The Second International Human Genome Editing Summit. He
was charged with illegal medical practices and faced 3 years of prison time. As mentioned in the
ethical debate before, many deemed his work as unethical, with far too great risks than benefits.
An article in the Journal of Law and the Biosciences called for stricter regulation policies in
China surrounding the scientific community. Prior to Dr. He Jiankui’s experiments, regulation on
gene editing was quite lax but has since improved with the Chinese Ministry of Education
requiring checks on gene editing research in universities. New regulations were also introduced
by the government for gene editing in which clinical trials needed to be approved by a National
Medical Ethics Committee. Additionally, in 2019, a “moratorium” (a temporary prohibition of
activity) was created for the clinical application of the use of CRISPR gene editing in human
germline research because permanent changes could be passed down through the generations.
The CRISPR/Cas9 system can potentially be used for many applications that can benefit the
world, but the scientific community insists that it is far too soon to implement into human
embryos. With the recent events, many new regulations and laws are expected to be
introduced as the genome editing technology continues to make advancements.

Molecular Biology DNA Notes

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