AMMAN
— Genetic diseases, like
sickle cell anemia, have long been thought of as
permanent. But a new technology with the ability to change genes themselves is
opening up new doors and allowing doctors to treat diseases once thought
incurable.
اضافة اعلان
Researchers from
a
study conducted by Vertex Pharmaceuticals and CRISPR Therapeutics have used the CRISPR gene-editing technique, which
utilizes the protein Cas9 to locate the desired portion of DNA before
removing, adding, or modifying sections of DNA sequences in the genome.
CRISPR (which
stands for clustered regularly interspaced short palindromic repeats) is a
group of DNA sequences found in genomes of prokaryotic organisms such as
bacteria. These sequences play a crucial role in the defence system of bacteria
and have since been modified as this new technology for gene editing.
Delivering the Cas-9 protein allows for the targeting of specific segments of
genetic code and the ability to edit or cut DNA sequences at specific
locations.
Genes are made up of DNA. DNA, or
deoxyribonucleic acid is a molecule that contains the biological
information that makes every living thing unique. However, an abnormality in a
single gene can cause lifelong illnesses such as Sick Cell Anemia and Cystic
Fibrosis.
CRISPR-Cas9 has
not only opened the door for possible cures to genetic diseases but also for the
early prevention and treatment of these diseases as well.
CRISPR’s
potential is currently being
studied with a few diseases, including cancer, neurodegenerative
disorders, and blood disorders, according to a statement released by the two
companies. The genetic engineering technology has already made it to various
clinical trials and has shown promising outcomes, the statement added.
Today, we have
far more knowledge of specific genes involved in these diseases and how they
work. Using this information, scientists can repair the damage done to the
gene, insert new genes, or disable harmful ones.
The trials performed
by Vertex Pharmaceuticals and CRISPR Therapeutics have found that sickle cell
anemia, a genetic blood disorder, is one of many conditions that have responded
well to gene editing treatment. Clinical trial patients treated with CRISPR no
longer needed blood transfusions and showed significant improvement of sickle
cell anemia symptoms.
Another blood
disorder called Beta thalassemia has also shown improvement in trials using CRISPR
techniques, according to the study. Beta thalassemia, an inherited blood
disorder, occurs when the body cannot make hemoglobin, the part of red blood
cells that carries oxygen, properly. It can result in anemia and other medical
issues.
As this
technology is relatively new, many diseases are still being examined in
clinical trials. Researchers,
in the statement, said that they are hopeful about this technique as it
has already shown promise in many diseases early on.
The majority of
these diseases have been thought to be life-threatening in the past. However, as
scientists continue to make advancements in medicine, many illnesses may be
prevented before the need for a cure.
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