Uploaded by Nakia Emmanuel

Gene Therapy essay

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INTRODUCTION
Gene therapy is a technique which is on the experimental level that uses
genes which are a sequence of nucleotides in a person’s DNA to prevent
and treat different diseases. This technique, within time can help many
doctors treat an illness in the future by injecting genes into the cells of a
patient without the use of surgery and drugs. HIV (human
immunodeficiency virus) attacks the T helper cells of the body's immune
system, resulting in AIDS if this goes untreated. Gene therapy can be
used against multiple targets to engineer protection against HIV
infection of cells, to purge the virus from infected cells and enhance
immune defenses that attack HIV.
IS GENE THERAPY A NEW HOPE FOR COMBATING HIV?
Gene therapy is a new hope for combating HIV as there are currently no
cures towards this virus and its prominence grows everyday as multiple
people become affected. Gene therapy is considered a new hope to
combat this virus because of the multiple approaches of testing being
done. Some of these approaches include: The replacement of the
mutated gene which may cause disease with a copy of the healthy gene,
deactivating a mutated gene that is functioning inadequately, as well as,
gene editing techniques. Out of these, gene editing is one of the most
promising strategies that is being tested.
There has then been development of effective anti-HIV-1 transgenes.
For the very first time, many researchers have begun using a technique
which edits genes which is used to create cells which are resistant to
HIV infections and can target cells which are infected with HIV. They
were able to completely remove the HIV gene for the cell which is
infected which is shown by the drop in the cell’s overall rate of its
production of HIV. In cells which are not already infected, the therapy
has become part of their genome, which produces cells that are resistant
to infection for a prolonged period.
This technique which uses gene-editing has so far only been used on
cells which are contained in a laboratory dish, however, this study will
make us one step closer to a therapy which could be administered into
and injection which will work withing the human body. The scientists in
this study use a combination of two different modules. The first one is a
probe which consist of two pieces of so-called “guide RNA” (gRNA)
which delicately uncovers and homes in on the two ends of the HIV
genome and the second is an enzyme called Cas9 which removes the
disease, causing material and reconnects the two ends of the human
DNA. This is the study to show that having a combination of an HIV
gene locator and remover can remove the HIV genes and also have it be
repaired the genome of the infected HIV cells with HIV infected cells
treated in laboratories and can reduce the infection production. It also
indicates that gene therapy is a safe technique and will not affect non
infected DNA.
Due to the advancements not just in gene editing but as well as the other
approaches, gene therapy should be able to be used as a cure for not just
HIV but other genetic and chromosomal diseases as well. This is a hope
of the future because although many advancements and breakthroughs
have been made, many testing trials are to still be undergone to make
gene therapy a common procedure.
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