Friday, April
14, 2023 NEWS TODAY Issue
#10
Latest news and bulletin updates
Jacob Rodriguez
CRISPR Technology
Revolutionizes Medicine
The latest updates
In recent years, the implementation of
CRISPR systems in clinical practices
seems to become more and more possible
as there has been extensive research on
CRISPR technology to test and improve
the gene manipulating technique. The
discovery of CRISPR technology has
revolutionized the field of gene editing,
opening up new possibilities for treating
genetic disorders and improving human
health. This powerful tool has been used
in a wide range of applications, from basic
research to agriculture, and now, it is
starting to find its way into clinical
practice. CRISPR: [Link]
CRISPR (Clustered Regularly Interspaced
Short Palindromic Repeats) is a gene-
editing tool that allows scientists to
modify DNA sequences in a highly
precise manner. It works by using a Clinical trials are already underway to test A group of scientists in 2020 composed a
special enzyme called Cas9, which can be the safety and efficacy of using CRISPR to study using CRISPR technology to find
programmed to cut DNA at specific treat genetic disorders. For example, a whether this gene therapy can be used to
locations. Once the DNA is cut, scientists clinical trial in China is testing the use of treat patients with sickle cell anemia
can insert or delete genes, which can CRISPR to treat beta-thalassemia, a genetic (SCD) and transfusion-dependent β-
potentially cure genetic diseases or disorder that affects the production of red Thalassemia (TDT). SCD and TDT are
improve human health. blood cells. If successful, this treatment both very severe monogenetic diseases
could provide a cure for patients with beta- that can become fatal at worst.
One of the most promising applications of thalassemia and other genetic disorders.
CRISPR technology is in the treatment of Two subjects were tested in this study,
genetic diseases. Many genetic disorders, Another potential application of CRISPR one had SDT and the other had TDT. The
such as cystic fibrosis and sickle cell technology is in cancer treatment. Cancer is scientists used CRISPR-Cas9 arrays to
anemia, are caused by mutations in a often caused by mutations in multiple correct a gene that had mutated and
single gene. By using CRISPR to edit the genes, and CRISPR could be used to target transplanted it back into the subject with
faulty gene, scientists can potentially cure these mutations and prevent cancer cells SCD. This gene functions in producing
these diseases at the source from multiplying. While this application is hemoglobin, a protein that carries oxygen
still in the early stages of development, it in the blood, and for patients with SCD,
holds great promise for the future of cancer the gene mutates and causes red blood
treatment. cells to break down. For TDT, a different
gene that is responsible for producing
Despite the potential benefits of CRISPR beta-globin was targeted. Beta-globin in
technology, there are also concerns about patients with TDT contain mutations that
its safety and ethical implications. Some prevent the production of healthy red
worry that using CRISPR to edit human blood cells. CRISPR technology was
genes could lead to unintended again used to correct these mutations and
consequences or even create new diseases. then transplanted the gene back into the
Others are concerned about the possibility patient.
Follow our CRISPR Instagram account
@crisprftw to stay up to date on CRISPR
of using CRISPR for non-medical
research! purposes, such as enhancing human
intelligence or physical abilities.
The scoop of the day The scoop of the day The scoop of the day
The latest updates The latest updates The latest updates
Mirjam Nilsson Mirjam Nilsson Mirjam Nilsson
Local man tries to teach pet rock how to Cat burglar caught stealing from local Man creates life-sized replica of the
roll, ends up with a stationary friend. pet store, claims he was just trying to Titanic out of toothpicks, sinks in his
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NEWS TODAY
After the treatment using CRISPR-Cas9
gene therapy, both patients had normal
levels of healthy red blood cells, high levels
of hemoglobin, and the patient with SCD
had no vaso-occlusive episodes which are
painful complications that occur when
sickled red blood cells block blood flow and
deprive tissues of oxygen. This was a very
good sign that the patients had been cured of
their diseases, but there were some potential
side-effects to the treatment during the
process.
The patient with TDT experienced
pneumonia and 31 other adverse events,
though all of which were not too severe or
life-threatening. The patient with SCD,
however, had 114 adverse events within the
16.6 months that she was monitored. Only 3
of these events were considered serious but
were all resolved with treatment. There was
no evidence of off target editing but there
were some potential sites that were Marcus Bernard
identified computationally using sequence
similarity. References: Local Man Tries to Teach Rock
Overall, this study is a potential breaking Frangoul, Haydar et al. “CRISPR-Cas9 How to Roll, Ends Up with a
point in medicine as patients with SCD and Gene Editing for Sickle Cell Disease Stationary Friend
TDT can be cured using CRISPR gene and β-Thalassemia.” The New
therapy, but careful implementation of this England Journal of Medicine, 2020, Blah Blah Blah...
technology is paramount. [Link]
6/NEJMoa2031054?
articleTools=true.
Abudayyeh, Omar, and Jonathan
Gootenberg. “CRISPR Diagnostics |
Science.” Science, 28 May 2021,
[Link]
science.abi9335.
Sohail, Muhammad et al.. “Methodologies
in Visualizing the Activation of
CRISPR/Cas: The Last Mile in
Developing CRISPR-Based
Diagnostics and Biosensing – A
Review.” Analytica Chimica Acta,
Elsevier, 25 Jan. 2022,
As with any new technology, it is important [Link]
to carefully consider the potential risks and e/article/pii/S000326702200112X.
benefits of using CRISPR in clinical
practice. However, the potential benefits of
this technology are truly remarkable, and it
offers new hope for patients with genetic
diseases and other health conditions. With
continued research and development,
CRISPR could someday become a routine
part of clinical practice, helping to improve
human health and transform medicine as we
know it. With the careful implementation of
CRISPR gene therapy in clinical trials
regarding genetic disorders and diseases,
people will not have to suffer from such
diseases anymore.
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