Gene therapy in treating sickle cell anemia disease
Sickle cell anemia disease or in short known as SCD is an inherited and serious condition affecting the body's blood and various organs. This disease can give effects to the red blood cells that can cause sickle-like episodes that cause pain and other symptoms. People with SCD are...
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| Main Authors: | , , , |
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| Format: | Book Section |
| Language: | English |
| Published: |
Penerbit UTHM
2022
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| Subjects: | |
| Online Access: | http://eprints.uthm.edu.my/7508/1/2.pdf http://eprints.uthm.edu.my/7508/ |
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| Summary: | Sickle cell anemia disease or in
short known as SCD is an inherited and
serious condition affecting the body's
blood and various organs. This disease can
give effects to the red blood cells that can
cause sickle-like episodes that cause pain
and other symptoms. People with SCD are
generally well in between episodes of
sickling which may cause a long term of
complications. Certain situations may
trigger sickling, like cold, low oxygen levels,
infection or lack of fluid in the body
(dehydration). This complication of disease
can be prevented from the root with a good
treatment. Therefore, early diagnosis and
specialized treatment for SCD are
recommended from doctors. Based on the
previous research, the method used in
treating this disease is stem cell
transplantation that can be found in bone
marrow. Actually, every transplant of bone
marrow has risks and bad effects on the
patient. It can give an infection to the
immune system like attack the new stem
cells that will cause failure to the transplant.
Moreover, once the stem cell of the donor
doesn't match with the recipients, the new
immune system cell of donors would attack
a few organs of recipients. So, these
complications are very dangerous to the
donors and recipients. Based on the
medical biotechnology that has a lot of
benefits, this research will focus on treating
disease by gene therapy with CRISPR/Cas9
as a method and an adeno-associated virus
(AAV) as a vector. In this research which is
based on application of biotechnology, it
will be focusing on gene therapy. Gene
therapy is also one of the best treatments
for this disease. The main reason for
patients with sickle cell is causing the
disorder of the adult hemoglobin gene, not
the fetal hemoglobin gene during the gene
mutation. So, this gene therapy in the sickle
cell operates by growing the production of
the selected gene by restoring the transition
performance again to fetal hemoglobin,
which does not sickle, and subsequently
decreasing sickle hemoglobin and thereby
increasing fetal hemoglobin. |
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