c




 


 

 is a scientific technique, by which a segment of nucleotide sequence or
gene is inserted into an individual's cell for the purpose of treating diseases× The main principle
behind gene therapy is to restore the normal functioning of cells and tissues by replacing
abnormal or mutated genes× In simple terms, it is a method of correcting defective genes× There
are several ways of implementing gene therapy; inserting a normal gene in the genome to replace
a defective gene and changing the regulation for expression of a specific gene× Another method
of gene therapy is to apply selective reverse mutation so that the defective gene returns to its
original form×

 

 can be broadly classified into two types:

One is the 
 

 and the other is the



 
 

× In somatic cell gene therapy, the somatic cells are targeted for gene replacement,
whereas in the reproductive cell gene therapy, the defective gene lies in the reproductive cells
that are replaced by the correct gene× The alterations made in the genetic makeup of the somatic
cells is corrective only for the patient× This change is not inherited by the treated person's
offspring× However, in case of germline gene therapy, the changes are passed on to the
descendants of the treated individual× 


p
  






 when the concept of gene therapy first surfaced× For them tinkering with the genetic
constitution of human beings was equivalent to 
, and this they perceived as being
sacrilegious! On the other side was the scientific community, abuzz with excitement at the
prospect of being able to wipe certain 

 
 entirely from the human
gene pool× Although the term  

 was first introduced during the , the
controversy about the rationality of this line of treatment still rages on× In the center of the debate
lie the gene therapy pros and cons that derive opinions from religious, ethical and undoubtedly,
political domains×

In order to be able to appreciate people's concerns about gene therapy there is need to first
understand the pros and cons of gene therapy×  

 is thought of by some people as an
terrific discovery that could greatly improve the human population and also give people with
certain disabilities some glimmer of hope× And on the other side of the coin there is some people
that think we have no business playing God× They feel that playing God may possibly cause
devastating effects to the human gene pool and may also led to misuse×



½
 


I think the pros all seem like very good ideas and gene therapy is a successful experiment×The
single most factor that gives gene therapy its edge is its incredible therapeutic potential×
 

 absolutely is the key to solving many mysteries about genetic disorders and potentially
can help to develop cures×

Ñc
 


















  






 
 
 
c  This fate that Watson is talking about is contained in
our genes, and deals with a new technique, gene therapy× Gene therapy is revolutionizing the
world of medicine× Many physicians are predicting that in twenty years gene therapy may change
the practice of medicine from a treatment-based to a prevention-based practice× Our future is
locked away inside of our genes× Gene therapy is unlocking these doors× Researchers are starting
to move away from developing new drugs, and towards finding an ultimate solution× That
solution is to use gene therapy as a treatment for many genetic diseas es× Researchers hope that
in the coming years, every genetic disease will have gene therapy as its treatment× Gene therapy
could be the last therapy that the human race will ever need×

‰ The human race has always been under the continuous onslaught of diseases× As we find
cure for some of the diseases, we are attacked by new and more virulent forms of germs×
Although such diseases can be cured through medicines, genetic disorders have no cure
unless the defective gene is replaced by the correct one and this is what gene therapy
aims at× Moreover, by targeting the reproductive cells, such defects can be got rid of for
good× People suffering from genetic disorders like ½

 
 ! 


 and  
 are some of those whose only hope for cure is gene
therapy×

‰ There have been numerous



 "


  in gene therapy of disorders
like inherent blindness, cancer, myeloid disorders, and sickle-cell
disorders× #



 focus on re-engineering lymphocytes to target and
attack cancer cells, or using tumor-suppressing genes to reduce number and size of
tumors×

‰ There are 
  

 
 that may be cured by gene therapy of which include
Rubinstein-taybi syndrome, partial epilepsy, cataracts, prostate cancer , male infertility,
Alzheimer¶s, schizophrenia, usher syndrome, and maternal acute fatty liver of pregnancy×
All of these disorders had there genes identified in 1995× ecause of its accuracy, gene
therapy has the potential to eliminate cancer cells without damaging normal, healthy
tissue× Furthermore, cancer gene therapies may provide alternatives when a disease does
not respond to other older treatments×

‰ Though the technology of 
   

 is not so advanced in the present
scenario, it has already widened the scope of medical science× As all genetic disorders
and most of the chronic diseases involve malfunctioning of the genes, gene therapy is a
promising technique for treatment of such severe health conditions× Genetic researches
based on human gene therapy are ongoing to discover treatment options for chronic
medical conditions such as ‘eart disease, cancercystic fibrosis and 3
× Nowadays, private
 grants as well as government grants are provided so as to encourage studies on human
gene therapy×

‰ -

 have fears that existing conditions will continue to be passed on to future
generations×  

 offers these people the hope that hereditary disorders will one
day be eradicated× People with existing conditions also may benefit from current and
future developments in the research of gene therapy× Gene therapy is the only existing
option for one day curing and preventing future genetic disorders×


# 



Meanwhile, the cons are a bit 

 though× Some people think that we will get burnt
trying to play God while others think this is a great advancement for man kind×
One reason for unacceptance may be that   

 may be used for the enhancement or
modification of human capabilities× If this were feasible, the standards of a normal human being
would be changed× Even if some small countries experimented with gene therapy, could they
develop unstoppable armies? If gene therapy was done to a certain extent could it alter the
human gene pool for good? And also would this form of treatment be a luxury only for the rich
which could very well make the rich, richer and make the poor, poorer×


$

A negative aspect of gene therapy includes that it is still largely uncharted territory× Most of the
science behind it depends on theories and ideas rather than solid facts×
A Although the most popular choice as a 
, using 
 for the purpose is not
foolproof× There is a specific point within the host genome where the correct gene should
be introduced× There is no guarantee that the viral enzyme that is responsible for this step
will be able to introduce the correct gene at the specific point in the host chromosome× In
 case, there is an error in this process, it would result in error in the genetic makeup of the
cell and can result in 


×

A There are many potential side effects of using 


, some of which include


%

 
", 



    immune system and at times,
&×

A A more recent technique of ‘uman gene t‘erapy is to introduce the normal gene directly to
the target cell without using vectors× Though this method sounds comparatively easy, it is
not applicable to all types of target cells× Another disadvantage of this method is that it
requires 


  '( ×

A Moreover, the body's   may destroy the vector as it may perceive the
carrier as a foreign body× Then there are problems with introducing therapeutic DNA and
the rapidly dividing nature of certain cells that are hurdles in gene therapy providing long
term benefits to patients× Due to this reason a patient may need to undergo multiple gene
therapy treatments× ut with this again comes the problem of the immune system× Once
the immune system is triggered by a foreign body, it attacks the foreign body more
aggressively when it invades the body next time×

) 
$

Ethical as well as legal issues are concerns when discussing gene therapy×Given the technology
involved, it is obvious that  



 will be expensive× It will be just the rich
who would be able to afford its benefits× This gives rise to the refrain that gene therapy will
make the rich, richer and the poor, poorer×We know the potential of reproductive gene therapy×
The scope of this line of treatment triggers the fear of eugenics - a term that denotes creation of a
superior race, the idea that media has tried to embody in the concept of designer babies× The cost
of gene therapy is high at this time and could be a concern for families with that need lifelong
treatment and do not have health care×






*$

-


 of man is absolutely unacceptable by those with strong religious

beliefs× According to them altering genes is similar to tinkering with nature× It's like questioning
God's will or in other words, 
× They feel that playing God may possibly cause
devastating effects to the human gene pool and may also led to misuse×
$




Invasion of privacy is also a concern for many× Insurance companies could make it mandatory to
have genetic screening before a policy before they issue a policy× This could cause
discrimination to families with genetic diseases× Or potential employers could question a person's
genetic background and pass them up for a promotion or job×

* 



The most important concern is the

 

× It is still in the developmental stage×
Finding the precise location of the defected gene is still under research× Any miscalculation
could cause a risk of a new disease such as cancer to form×

 
%


A 
+


,"
 

 is the short-term effectiveness of the therapeutic gene after
introducing into the target cell× It is the dividing cells present in the body that prevents the
expression of therapeutic genes× After inserting the foreign therapeutic gene into the target cell, it
is necessary to regulate the autoimmune responses of the body× Human gene therapy is less
effective for 
" (presence of many defective genes)×

#


 




 
The -
 '
 

 .-' / has not yet approved any human gene therapy
product for sale× Current gene therapy is experimental and has not proven very successful in
clinical trials× Little progress has been made since the first gene therapy clinical trial began in
× In , gene therapy suffered a major setback with the death of 18-year-old Jesse
Gelsinger× Jesse was participating in a gene therapy trial for ornithine transcarboxylase
deficiency (OTCD)× He died from multiple organ failures 4 days after starting the treatment× His
death is believed to have been triggered by a severe immune response to the adenovirus carrier×

Another major blow came in January ë0, when the FDA placed a temporary halt on all gene
therapy trials using retroviral vectors in blood stem cells× FDA took this action after it learned
that a second child treated in a French gene therapy trial had developed a leukemia-like
condition× oth this child and another who had developed a similar condition in August 2002 had
been successfully treated by gene therapy for X-linked severe combined immunodeficiency
disease (X-SCID), also known as "bubble baby syndrome×"

-'  1
 * -

 
 # .1*- #/ met at the end of
February 2003 to discuss possible measures that could allow a number of retroviral gene therapy
trials for treatment of life-threatening diseases to proceed with appropriate safeguards× In 

ë0 the FDA eased the ban on gene therapy trials using retroviral vectors in blood stem cells×


-
 



The 
 

 is great but, compared to its promise, the results to date are still
quite limited× However, the benefits of gene therapy are believed to be on the near horizon× Gene
therapy is one of the hottest areas of medical research today× (And gene therapy companies have
been among the hottest in the stock market×)
The remarkable advances in, including the 

+, have opened new
doors for the exploration of  

× New technologies are needed to speed the progress of
gene therapy×Gene therapy will play an increasingly important and prominent part in medicine in
the decades to come×

Gene therapy is an exciting new approach that is just making the news×Gene therapy will change
the field of medicine from what it is today× As s cientists discover more genes and their
functions, the potential of this treatment is limitless× Our genome is the "
 of our body×
m










  × As researchers start to understand this blueprint,
our lives will be forever changed× We now know our fate is indeed in our genes×

#
To take a stand on  

 means accepting both the pros and cons× It is clear gene therapy
will have an affect on the future of genetics× I think with careful planning and regulation gene
therapy will become the "medicine" of the near future× The concern of interfering with evolution
is misconceived×