GENE
THERAPY – SCI & TECH
News: US approves two gene therapies for
sickle cell disease
What's
in the news?
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The U.S. Food and Drug Administration
(FDA) approved a pair of gene therapies for sickle cell disease, including the
first treatment based on the breakthrough CRISPR gene editing technology.
Key
takeaways:
●
Gene therapy is an experimental technique that caters to patients with such diseases.
Gene
therapy:
●
Gene therapy is a technique which involves
the replacement of defective genes with healthy ones in order to
treat genetic disorders.
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It is an artificial method that introduces DNA into the cells of the human body.
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The first gene therapy was successfully
accomplished in the year 1989.
Procedure:
●
The cell
with the defective gene is injected with a normal gene which helps in the
normal functioning of the cell.
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This technique is employed mainly to fight
against the diseases in the human body and also to treat genetic disorders.
●
The damaged proteins are replaced in the
cell by the insertion of DNA into that cell. Generally, improper protein
production in the cell leads to diseases.
●
These diseases are treated using a gene
therapy technique.
●
For example, cancer cells
contain faulty cells which are different from the normal cells and have
defective proteins. Hence, if these proteins are not replaced, this disease
would prove to be fatal.
Types
of Gene Therapy:
1.
Somatic Gene Therapy:
●
This type usually occurs in the somatic
cells of the human body.
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This is related to a single person and the only person who has the damaged
cells will be replaced with healthy cells.
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In this method, therapeutic genes are
transferred into the somatic cells or the stem cells of the human body.
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This technique is considered as the best and safest method of gene therapy.
2.
Germline Gene Therapy:
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It occurs in the germline cells of the
human body. Generally, this method is adopted to treat the genetic, disease-causing-variations of genes which are passed
from the parents to their children.
●
The process involves introducing a healthy
DNA into the cells responsible for producing reproductive cells, eggs or
sperms. Germline gene therapy is not legal in many places as the risks outweigh
the rewards.
Application
of Gene Therapy:
●
It is used in the replacement of genes
that cause medical ill-health.
●
The method generally destroys the problem
causing genes.
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It helps the body to fight against
diseases by adding genes to the human body.
●
This method is employed to treat diseases
such as cancer, ADA deficiency, cystic fibrosis, etc.