Gene therapy

Rosemarie Ramones
BSIT-1B
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What is Gene Therapy?
 A wide range deseases such as:

Cystic fibrocis Diabetes

Cancer
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Heart desease Aids

Hemophilia
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 Gene Therapy is an experimental technique

developed in order to treat these disease.
 Gene Therapy is the introduction removal or
alternation of genetic material in order to prevent
or treat a deseases
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How does gene therapy work?

 Ideally, gene therapy may someday allow doctors to treat

disorders by inserting or replacing genes into a patient's
cells instead of through surgery or drugs (U.S. National
Library of Medicine, 2020). There are various approaches
in performing gene therapy, including:
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 a. Replacing a disease-causing mutated gene

with a healthy copy of the gene
 b. Inactivating or "silencing" a mutated gene
or a gene that is not functioning properly
 c. Introducing a new gene into the body to
help counteract or fight the disease
 d. Correcting the sequence of a mutated gene
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Replacing A Mutated Gene (GENE REPLACEMENT) In

order to replace a problematic or mutated gene:
*A functional and healthy gene will be inserted to a
viral vector
*The viral vector will carry and place the healthy gene
on the site of mutation.
*The mutated non-functional gene will be "spliced" or
cut and removed
*The healthy gene will be attached and becomes a
part of the final DNA strand.
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GENE SILENCING OR GENE
KNOCKDOWN
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This method aims to repress or turn off the function of a mutated

gene which prevents the cells from producing disease-causing
proteins. Gene silencing reduces the expression of a disease
causing gene (Hood, 2004; Mocellin & Provenzano, 2004). It can be
done by:

a. inserting a repressor aka silencer gene that reduces or prevents

the expression of the mutated gene

b. b. editing the gene to make it impossible for it to produce the

protein it codes for
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Although gene silencing is often considered as the same thing as

gene knockdown, this is debatable because some say that when
genes are knocked out, they are often completely removed or
erased from the genome in order to stop their expression (Hood,
2004; Mocellin & Provenzano, 2004).