INSUBCONTINENT EXCLUSIVE:
CRISPR, the revolutionary ability to snip out and alter genes with scissor-like precision, has exploded in popularity over the last few
years and is generally seen as the standalone wizard of modern gene-editing
However, it not a perfect system, sometimes cutting at the wrong place, not working as intended and leaving scientists scratching their
Well, now there a new, more exacting upgrade to CRISPR called Prime, with the ability to, in theory, snip out more than 90% of all genetic
diseases.
Just what is this new method and how does it work? We turned to IEEE fellow, biomedical researcher and dean of graduate education
at Tuft University school of engineering Karen Panetta for an explanation.
How does CRISPR Prime editing work?
CRISPR is a powerful genome
It utilizes an enzyme called Cas9 that uses an RNA molecule as a guide to navigate to its target DNA
It then edits or modifies the DNA, which can deactivate genes or insert a desired sequence to achieve a behavior
Currently, we are most familiar with the application of genetically modified crops that are resistant to disease.
However, its most
promising application is to genetically modify cells to overcome genetic defects or its potential to conquer diseases like cancer.
Some
applications of genome editing technology include:
Genetically modified mosquitos that can&t carry malaria.
In humans, &turning on& a gene
that can create fetal type behaving cells that can overcome sickle-cell anemia.
Of course, as with every technology, CRISPR isn&t perfect
It works by cutting the double-stranded DNA at precise locations in the genome
When the cell natural repair process takes over, it can cause damage or, in the case where the modified DNA is inserted at the cut site, it
can create unwanted off-target mutations.
Some genetic disorders are known to mutate specific DNA bases, so having the ability to edit these
bases would be enormously beneficial in terms of overcoming many genetic disorders
However, CRISPR is not well suited for intentionally introducing specific DNA bases, the As, Cs, Ts and Gs that make up the double
helix.
Prime editing was intended to overcome this disadvantage, as well as other limitations of CRISPR.
Prime editing can do multi-letter
base-editing, which could tackle fatal genetic disorders such as Tay-Sachs, which is caused by a mutation of four DNA letters.
It also more
I view this as analogous to the precision lasers brought to surgery versus using a hand-held scalpel
It minimized damage, so the healing process was more efficient.
Prime editing can insert, modify or delete individual DNA letters; it also
can insert a sequence of multiple letters into a genome with minimal damage to DNA strands.
How effective might Prime editing be?
Imagine
being able to prevent cancer and/or hereditary diseases, like breast cancer, from ever occurring by editing out the genes that are makers
Cancer treatments are usually long, debilitating processes that physically and emotionally drain patients
It also devastates patients& loved ones who must endure watching helpless on the sidelines as the patient battles to survive.
&Editing out&
genetic disorders and/or hereditary diseases to prevent them from ever coming to fruition could also have an enormous impact on reducing the
costs of healthcare, effectively helping redefine methods of medical treatment.
It could change lives so that long-term disability care for
diseases like Alzheimer and special needs education costs could be significantly reduced or never needed.
How did the scientific community
get to this point — where did CRISPR/prime editing &come from?&
Scientists recognized CRISPR ability to prevent bacteria from infecting
more cells and the natural repair mechanism that it initiates after damage occurs, thus having the capacity to halt bacterial infections via
Essentially, it showed adaptive immunity capabilities.
When might we see CRISPR Prime editing &out in the wild?&
It already out there! It
has been used for treating sickle-cell anemia and in human embryos to prevent HIV infections from being transmitted to offspring of HIV
parents.
So, what next?
IEEE engineers, like myself, are always seeking to take the fundamental science and expand it beyond the petri dish
to benefit humanity.
In the short term, I think that Prime editing will help generate the type of fetal like cells that are needed to help
patients recover and heal as well as developing new vaccines against deadly diseases
It will also allow researchers new, lower cost alternatives and access to Alzheimer like cells without obtaining them post-mortem.
Also, AI
and deep learning is modeled after human neural networks, so the process of genome editing could potentially help inform and influence new
computer algorithms for self-diagnosis and repair, which will become an important aspect of future autonomous systems.
