Blood
is a fluid or a semi-liquid material that flows within the human body,
circulating around from our head to our toes and is crucial for the survival of
humans. Blood is composed of a diverse a unique mixture of cells and
biochemical molecules and each one performs a specific function which is essential
to the survival of a human. These blood related molecules are crucial for
multiple life sustaining activities like the transportation of oxygen O2,
healing and repairing of wounds, translocation of sugar molecules etc. Some of
these molecules (cells) are often unable to perform their function because of
certain genetic mutations resulting in to malformation of cells and ultimate
reduction in activity of those cells which threatens the survival of a human.
THE DISEASE:
Sickle
cell and thalassemia are among the most common blood disorders and are cause d due
to a specific genetic mutation in genes encoding for haemoglobin (molecule transporting
oxygen). According to a detail study published in LANCET in 2015-16 a total 4.5
million peoples are affected by the sickle cell where as more than 228000
people are affected by thalassemia disease and annual deaths cuased by the 2 diseases
is around 130,000 annual. This implicates the severity of this disease all over
the world, in addition to this patients who continue to live with these
disorders are faced with severe problems including repeated painful
blood-transfusion, feeling exhausted, weak, pale skin colour, oxygen deprivation
and more.
OLD SOLUTIONS:
Treatment
of this disease often involves severe blood transfusions, intensive care,
medicines and in severe cases stem cell therapy which is often faced with
multiple challenges including scarcity of donors, rare donor cross match, and chances
of graft rejection, which results in severe outcomes.
NEW SOLUTION:
Owing
to the risk and rare possibility of success related to previous treatments need
for modern approaches is inevitable, one the most prominent approach for curing
genetic disorders is the use of CRISPR-gene editing technique. A detailed study
is being performed by CRISPR-Therapeutics and US pharmaceutical giant Vertex
Pharmaceuticals in US, the study involves the application of a detailed process
using gene editing called the “CTX001” which makes some specific point cuts in
to the DNA for the alteration of certain genes and triggering haemoglobin production.
THE PROCESS:
The
study is currently being carried out at the Sarah Cannon Research Institute in the US state of Tennessee where
many patients are currently enrolled for the trial. One of the very first
patient enrolled in the trial has recently being evaluated for possible improvements
in health conditions and the results are astonishing.
The
trial itself involves using the CRISPR gene editing process to clip of certain “SWITHC-OFF”
genes present in the host stem cells responsible for the turning off the production
of fetal haemoglobin or the HbF resulting
in to the production of fetal-haemoglobin molecules. The HbF itself is a kind of haemoglobin
molecule that exists in the body during fetal stage and is later replaced by
the adult version of haemoglobin but has almost similar tendency of carrying
out all normal functions.
THE RESULT:
Recently
results for the initial phase of the research were released by the team which
states that the blood analysis of Victoria
Gray (the 1st patient) an approximate 47% of stable fetal haemoglobin
(HbF) in the blood with nearly 95% of them carrying the edited version which is
beyond expectations and a huge success.
Dr. Jeffrey Leiden, Chairman and
CEO of the Vertex Pharmaceutical said:
“The
result are remarkable and shows the ability and effectiveness of CTX001 for
curing these severe blood disorders. With this great initial phase success we
are looking forward to work along with researchers, doctors, patients and thier
families in a close relationship in future, and we hope to soon expand the treatment
impact to cure other serious diseases such as the Duchenne muscular dystrophy
and myotonic dystrophy type 1.”
 |
| Dr. Jeffery Leiden, CEO Vertex Pharmaceuticals |
CONCLUSION:
The
technology is proving itself to be a highly effective method to resolve many lethal
diseases which have been a major challenge for decades, after promising results
from animal trials to successful clinical trials the tech is now almost ready
for broader application, but the most necessary thing here is to get the public
ready for it and smooth, swift policy regulations by the regulatory authorities
and legislators to ensure in time arrival and application of the technique and
way before even this tech began to be obsolete for rising challenges.
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