Biology 100 � Section 005 Gene Therapy�s Potential in Curing Genetic Disease
Exploration of gene therapy's role in treating genetic disorders.
Eva Reed
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Biology 100 – Section 005
November 25, 2013
Gene Therapy’s Potential in Curing Genetic Disease
Based on the article Gene Therapy’s Potential in Curing Genetic Disease, critically analyze the
progress and challenges of gene therapy in treating genetic diseases. Discuss the potential
benefits of somatic gene therapy, including its successes and limitations. Additionally, evaluate
the ethical concerns surrounding germ-line gene therapy and its implications for future
generations. Your answer should provide a balanced assessment of the scientific, medical, and
societal considerations involved in gene therapy, and should be between 600-800 words in
length.
Biology 100 – Section 005
November 25, 2013
Gene Therapy’s Potential in Curing Genetic Disease
Based on the article Gene Therapy’s Potential in Curing Genetic Disease, critically analyze the
progress and challenges of gene therapy in treating genetic diseases. Discuss the potential
benefits of somatic gene therapy, including its successes and limitations. Additionally, evaluate
the ethical concerns surrounding germ-line gene therapy and its implications for future
generations. Your answer should provide a balanced assessment of the scientific, medical, and
societal considerations involved in gene therapy, and should be between 600-800 words in
length.
2
Gene Therapy’s Potential in Curing Genetic Disease
The completion of the Human Genome Project has opened the doors leading to
seemingly unlimited scientific possibilities. With a correct understanding of DNA, scientists can
identify specific sections that code for positive (or negative) results; they can enhance or
duplicate the positive sections and try to remove the ‘errors’. Genetically modified organisms
(GMO) typically refer to animals or food, but soon it could become commonplace for it to refer
to humans. Scientists have had great success in using genetics to improve the quality (and often
the quantity) of food that humans eat on a daily basis. Although controversial, many advocate
using this same technology on humans, particularly to cure them of disabling, genetic diseases.
Despite the dangers posed by human genetic modification, gene therapy has the
potential to cure the genetic diseases that plague humanity. This report will cover the history of
human gene therapy, its potential for curing disease, and the dangers presented by germ-line
gene therapy.
Human application of gene therapy was conceptualized in the early 1970s, by Theodore
Friedmann and Richard Roblin (1). They recognized the potential of gene therapy, but
cautioned that they did not have enough information to begin human testing. They proposed
that more research be done to make this a safe and effective treatment, and also advocated
“formulating a complete set of ethicoscientific criteria to guide the development and clinical
application of gene therapy techniques” (1).
It was almost twenty years later that the FDA approved the first gene therapy case in
the United States. Dr. William French Anderson oversaw the treatment of a four year-old girl,
Ashanti DeSilva, with ADA-SCID (Severe Combined Immunodeficiency); this genetic disease
Gene Therapy’s Potential in Curing Genetic Disease
The completion of the Human Genome Project has opened the doors leading to
seemingly unlimited scientific possibilities. With a correct understanding of DNA, scientists can
identify specific sections that code for positive (or negative) results; they can enhance or
duplicate the positive sections and try to remove the ‘errors’. Genetically modified organisms
(GMO) typically refer to animals or food, but soon it could become commonplace for it to refer
to humans. Scientists have had great success in using genetics to improve the quality (and often
the quantity) of food that humans eat on a daily basis. Although controversial, many advocate
using this same technology on humans, particularly to cure them of disabling, genetic diseases.
Despite the dangers posed by human genetic modification, gene therapy has the
potential to cure the genetic diseases that plague humanity. This report will cover the history of
human gene therapy, its potential for curing disease, and the dangers presented by germ-line
gene therapy.
Human application of gene therapy was conceptualized in the early 1970s, by Theodore
Friedmann and Richard Roblin (1). They recognized the potential of gene therapy, but
cautioned that they did not have enough information to begin human testing. They proposed
that more research be done to make this a safe and effective treatment, and also advocated
“formulating a complete set of ethicoscientific criteria to guide the development and clinical
application of gene therapy techniques” (1).
It was almost twenty years later that the FDA approved the first gene therapy case in
the United States. Dr. William French Anderson oversaw the treatment of a four year-old girl,
Ashanti DeSilva, with ADA-SCID (Severe Combined Immunodeficiency); this genetic disease
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University
National University
Subject
Biology