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I really knew nothing about gene therapy before reading about it in our textbook. Some

questions I had while reading about gene therapy were the following. If gene therapy becomes a

social norm to cure diseases such as cancer, will it be available to everyone? Will it be

affordable? How many vectors have been tested to ensure only the safest are used? The text

mentioned gene therapy took a turn for the worst when teenager Jesse Gelsinger passed away

due to complications after large numbers of adenovirus vectors were injected into his hepatic

artery causing a severe immune reaction. Are the health risks with gene therapy worth the

risk? One cool thing I learned about gene therapy is how the human immunodeficiency virus is

used as a vector for gene therapy. I would have never imagined HIV would be used for

something like gene therapy. This was impressive to read about! Cystic fibrosis would be a

good target for gene therapy because it’s a single gene disorder because the affected gene is

known and the target tissue, the lung can be accessed for treatment.

1. Some challenges scientists face is with gene delivery and activation. The therapy will

only work if a normal gene is delivered to many cells and is activated once

introduced. Another is the body’s immune response due to the vector selected and the

possible disruption of important genes in target cells.

2. Anyone prone to an illness caused by a disease that can be identified. Criteria usually

looked at are if the disease is identified and the gene can be cloned in laboratory

generally by treating a single gene. I don’t believe gene therapy should be used for

cosmetic purposes or to improve athletic performance.

3. Some challenges associated with gene therapy are the following, knowing the proper

routes for gene delivery for different types of disorders. Knowing the amount cells in an

organ or tissue need to express in order to alleviate the effects of a genetic disorder. How

much therapeutic gene product must be produced to provide lasting improvement of the

condition. As well as determining if gene therapy can treat diseases involving multiple

genes. Some ethical issues are the routes from which gene therapy can be

performed. There are currently two methods that are not approved due to ethical reasons,

germ-line therapy and enhancement gene therapy. Cost is another issue that hasn’t been

determined yet and will pose the question of what is the right price for a cure? As stated

in our book.

Gene therapy is surely a break-through medical science witnessed in the 21

century. Gene

therapy is the answer to those diseases which were previously considered untreatable and for

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I really knew nothing about gene therapy before reading about it in our textbook. Some questions I had while reading about gene therapy were the following. If gene therapy becomes a social norm to cure diseases such as cancer, will it be available to everyone? Will it be affordable? How many vectors have been tested to ensure only the safest are used? The text mentioned gene therapy took a turn for the worst when teenager Jesse Gelsinger passed away due to complications after large numbers of adenovirus vectors were injected into his hepatic artery causing a severe immune reaction. Are the hea ...
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