New Kid on the Block
– CRISPR
Akshat Jain MD MPH
CRISPR (Clustered Regularly
Interspaced Palindromic Repeats) is the most recent medical breakthrough, which
might very well be on the way to define the most remarkable medical discovery
of this century.
Imagine a time, where a person
could get a blood test, which would tell him the exact gene defect that are
causing his symptoms and pin point his “dis-ease”. He then takes his blood
sample to a “Gene Shop” where programmed segments of healthy DNA are “stitched”
in to his genome and injected back to him. A few pills and couple of months
later – Voilà!! He gets cured of what could be Alzheimer’s or Leukemia or Hemophilia,
Thalassemia, Sickle cell disease.
While the Tesla’s and Space X’s
of our time are busy to create the advanced intelligence “AI” and Hyperloop
etc. to transform society the medical technology of “Gene Editing” is quietly
making its way past animals to Human subject trials . CRISPR is the fancy abbreviation that brings Genomics – basic
science – protein synthesis – Medicine together, making another potential
successful payoff of investment made in genomics based research.
Gene editing tool – CRISPR
The technique may have
limitations in the form of a theoretical possibility of alteration in the
healthy gene segment inadvertently while the “tailoring” is underway, the outcome could be devastating. See the
gene segments are like musical notes on a chromosome, that the cell needs to
read to synthesize its protein sequence though its ribosomes that zip line down the pattern. Any
alteration in this pattern would produce a different final note, which may or
may not have been desired.
Although this application of CRISPR is far from taking to mainstream
medical interventions another innovative application of the process just
received the nod by the FDA this year. The application is to create what I like
to call “Super-Soldier Blood Cells”. Certain cells in our body are programmed
to kill. The offenders like bacteria, viruses, fungus etc. are usually the
targets for these T cell type of White Blood Cells (WBCs). With gene editing tools like CRISPR, these T cells are programmed to
identify their “targets” which are characteristic and remarkable only for cancer
cells in the body. Once these Modified T cells, called CART-T cells are created
in the laboratory, they are injected into the patients with cancer, and have
shown to work well by helping to get rid of the cancer cells from even the deep
crevices of the bone marrow.
This takes away from the fear of
inaccuracies of gene editing in live human body that may manifest in a
defective organ function. Nevertheless as more and more human trials get
completed, and safety data piles up, we are looking at a remarkable time in
human medical history.
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