GE1713

Topic Learnings
Name: Section: Date: 6/15/22

Instructions: Reflect on the topics that were previously discussed. Write at least three (3) things per topic
that you have learned. Answers must be written in complete sentences.

TOPIC LEARNINGS
Gene Therapy Gene therapy is the technique that modifies a person’s gene
(Definition and Principles)
to treat or cure disease. It involves the injection of genetic
material into cells to produce a useful protein. The principle has
three different strategies. First is by replacing a disease-causing
gene with a healthy copy of the gene. Second is by inactivating a
disease-causing gene that is not functioning properly. Lastly is by
introducing a new or modified gene into the body to help treat a
disease. A vector is a carrier that is genetically designed to convey
the gene. The DNA of adenovirus is not inserted into a chromosome
but instead, it is inserted into the nucleus of the cell.
Gene Editing Gene editing has the ability to change an organism’s DNA through a
Group of technologies that are used by scientists. Those
technologies allow genetic material to be added, removed, or
altered at Particular locations in the genome. It is accomplished by
the use of enzymes. Enzymes are the nucleases that have been
designed to target DNA sequences. It also allows the removal of
existing DNA and then the new DNA. The tool that is used in gene
editing will guide or serves as a template that matches either the
base pairs of the target sequence. It finds sequences that match,
the line up accordingly, and position where to cut.
CRISPR-Cas9 It is the most modern type of gene-editing tool and it has the most
a potent tool that can be used. It is a much better tool because of
its precision, efficiency and flexibility. Its system is a prokaryotic
immune system and the binding domain is based on short
repetitions of base sequences. The system of CRISPR-Cas9 is
composed of 3 main elements which are the Cas9, CRISPR RNA and
trans-activating crRNA. It also allows the deletion and insertion of
DNA because of that functioned with pinpoint accuracy. In short
it edits genes by precisely cutting DNA and then letting natural DNA
repair processes to take over.

10 Worksheet 1 *Property of STI

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 GE1713

Topic Learnings
Name Section: Date: 6/16/22

Instructions: Reflect on the topics that were previously discussed. Write at least three (3) things per topic
that you have learned. Answers must be written in complete sentences.

TOPIC LEARNINGS
Important Events in Gene In 1947, It described the possibility and potential of gene therapy
Therapy for correcting heredity diseases. In 1972 is the foundational
proposal and rationale for gene therapy. In 1980 is the the
premature and non-approved attempt at a clinical gene therapy. It
is said that there was no clinical benefit, and the human
experiments were performed without UCLA/ethics committee
approval. In 1990 is the first human gene transfer. In 2003 is the first
regulatory approval of a gene product. In 2012 is the first approved
gene therapy in Europe Glybera. In 2015 is FDA approves a first-of-
its-kind product for the treatment of melanoma. In 2016-2017, FDA
and European Commission approves multiple gene therapy product.

Diseases Being Studied for
Gene Therapy
Issues and Concerns
First is Leber’s Congenital Amaurosis (CLA), which is a genetic retina
disorder that causes visual impairment beginning in infancy.
Patients present usually with nystagmus, sluggish or near-absent
pupillary responses, severely decreased visual acuity, photophobia
and high hyperopia. Second is Duchenne Muscular Dystrophy (DMD)
It affects both skeletal and cardiac muscles and it is most commonly
inherited myopathy in childhood. With gene therapy trial on mice
and dog it has been approved because it have shown positive
results. The third is Hemophilia A&B, the therapy for this disease is
very expensive. With gene therapy, it has a slight improvement
genetically provides significant physical improvement.
It has an unwanted immune system reaction. Every body’s immune
system may see the newly introduced viruses as intruders and attack
them so it may cause inflammation and organ failure. It can also
target the wrong cells which can damage a cell, cause another illness
or diseases like cancer. It can also infect which is caused by the virus.
It is also possible to cause a tumor. If new genes is inserted in the
wrong spot of the DNA, it may cause tumor formation. Over a few
decades, this gene therapy has also faced several problems and
Because of lack of information it can cause long-term consequences.

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