Chapter  10.  Gene  Therapy  
 
  The   genes   in   the   body’s   cells   play   an   important   role   in   your   health   –   indeed,   a  
defective  gene  or  genes  can  make  someone  sick.  Recognizing  this,  scientists  have  been  
working  for  decades  on  ways  to  modify  genes  or  replace  faulty  genes  with  healthy  one  to  
treat,  cure  or  prevent  a  disease  or  medical  condition.  
 
  Cells  are  the  basic  building  blocks  of  all  living  things.  The  human  body  is  composed  
of   trillions   of   them.   Within   our   cells   there   are   thousands   of   genes   that   provide   the  
information   for   the   production   of   specific   proteins   and   enzymes   that   make   muscles,  
bones,  and  blood,  which  in  turn  support  most  of  our  body’s  functions,  such  as  digestion,  
making  energy  and  growing.  
 
  Sometimes  the  whole  or  part  of  a  gene  is  defective  or  missing  from  birth,  or  a  gene  
can  change  or  mutate  during  adult  life.  Any  of  these  variations  can  disrupt  how  proteins  
are  made,  which  can  contribute  to  health  problems  or  diseases.  
 
  In  gene  therapy,  scientist  can  do  one  of  several  things  depending  on  the  problem  
that  is  present.  They  can  replace  a  gene  that  causes  a  medical  problem  with  one  that  
doesn’t,  add  genes  to  help  the  body  to  fight  or  treat  disease,  or  turn  off  genes  that  are  
causing  problems.  In  order  to  insert  new  genes  directly  into  cells,  scientists  use  a  vehicle  
called  a  “vector”  which  is  genetically  engineered  to  deliver  the  gene.  Viruses,  for  example,  
have  a  natural  ability  to  deliver  genetic  material  into  cells,  and  therefore,  can  be  used  as  
vectors.  Before  a  virus  can  be  used  to  carry  therapeutic  genes  into  human  cells,  however,  
is  modified  to  remove  its  ability  to  cause  an  infectious  disease.  Gene  therapy  can  be  used  
to  modify  cells  inside  or  outside  the  body.  When  it’s  done  inside  the  body,  a  doctor  will  
inject   the   vector   carrying   the   gene   directly   into   the   part   of   the   body   that   has   defective  
cells.  
 
  In  gene  therapy  that  is  used  to  modify  cells  outside  the  body,  blood,  bone  marrow,  
or   another   tissue   can   be   taken   from   the   patient,   and   specific   types   of   cells   can   be  
separated  out  in  the  lab.  The  vector  containing  the  desired  gene  is  introduced  into  these  
cells.  The  cells  are  left  to  multiply  in  the  laboratory  and  then  injected  back  into  the  patient  
where  they  continue  to  multiply  and  eventually  produce  the  desired  effect.  
 
Approaches  to  Gene  Therapy  
 
1.  Gene  Modification  
  Researchers  have  used  the  following  methods  to  modify  defective  genes:  

•   Replacement  treatment:  Replacing  a  natural  gene  with  a  non-­natural  gene  through  

homologous  recombination.  

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 •   Modifier   gene   therapy:   Restoring   natural   function   to   a   defective   gene   through  
selective  reverse  mutation.  
•   Adjustment  of  the  expression  of  a  specific  gene.  

2.  Gene  transfer  method  

       There  are  3  physical,  chemical,  and  biological  methods  of  gene  transfer.  
 
3.  Gene  transfer  to  specific  cell  line  
       This  line  is  divided  into  2  general  categories  of  somatic  gene  therapy,  and  sex  cell    
       gene  therapy.  
 
4.  The  adoption  of  the  most  appropriate  genetic  engineering  (gene  injection)  
         Other  forms  of  genetic  engineering  include  gene  targeting  and  the  elimination  of    
         specific  genes  through  nuclease  engineering.  
 
Stem  Cell  Therapy  
 
A  stem  cell  therapy  is  any  treatment  that  uses  stem  cells  as  the  primary  way  of  curing  or  
reducing  the  severity  of  a  disease  or  disorder.  There  are  two  main  ways  stem  cells  can  
be  used:  
 
1.  as  a  transplant,  where  the  desired  stem  cells  are  harvested  either  from  the  patient  or  
a   donor   and   refined   or   modified   in   some   way   before   being   injected   or   grafted   into   the  
patient,  or  
 
2.  as  a  target  for  a  drug  or  other  biologic  where  the  drug  or  biologic  is  intended  to  activate  
a  desired  response  from  the  stem  cells  that  already  exist  in  the  patient’s  tissues  or  organs.  
 
Ethical  Dilemma  
 
The  possibility  of  destructive  embryo  research,  particularly  embryonic  stem  cell  research,  
presents   us   with   a   moral   problem   because   it   appears   to   bring   into   tension   two  
fundamental  moral  principles  that  people  esteem  very  highly:  one  principle  enjoins  the  
prevention  or  alleviation  of  suffering,  and  other  enjoins  us  to  respect  the  value  of  human  
life.  The  harvesting  and  culturing  of  embryonic  stem  cells  has  considerable  potential  to  
bring   about   remarkable   potential   benefits   in   the   way   of   alleviating   debilitating   medical  
conditions.  It  satisfies  the  first  principle  to  a  very  great  degree.  
 
  On   the   other   hand,   there   is   a   case   to   be   made   that   the   harvesting   of   human  
embryonic  stem  cells  violates  the  second  principle  in  that  it  results  in  the  destruction  of  
human   life   with   value   (i.e.   human   embryos).   Accordingly,   both   principles   apparently  
cannot   simultaneously   be   respected   in   the   case   of   embryonic   stem   cell   research.   The  
question  then  is  which  principle  ought  to  be  given  precedence  in  this  conflict  situation.  If  
weight   is   given   more   to   the   first   and   permit   destructive   embryonic   stem   cell   research  
because  of  its  remarkable  benefits?  Or  should  be  given  more  to  the  second  and  prohibit  

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destructive  embryonic  research  because  it  violates  respect  for  the  value  of  the  embryo  as  
the   very   beginnings   of   a   possible   human   life?   This,   at   bottom,   is   the   ethical   problem  
generated  by  destructive  embryo  research.    
 
Activity.  Video  Presentation  
 
A.  Transplant  Cells  not  Organs  
 
1.  Watch  TED  Talk  featuring  Susan  Lim  entitled  “Transplant  Cells  not  Organs”.  It  is    
       available  online  at  https://www.ted.com.  
 
2.  Answer  the  following  questions:  
 
  a.  What  is  the  main  topic  of  the  Dr.  Lim’s  speech?  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
 
  b.  What  do  you  think  prompted  scientists  like  Susan  Lim  to  inject  changes  that    
                             lead  to  development  of  medical  practice?  
         ______________________________________________________________  
           ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
 
  c.  In  your  opinion,  how  far  should  science  go  to  save  lives?  
                             ______________________________________________________________  
             ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
 
B.  The  Next  Species  of  Human  
 
1.  Watch  TED  Talk  featuring  Juan  Enriquez  entitled  “The  Next  Species  of  Human”.  It  is    
       available  online  at  https://www.ted.com.  
 
2.  Answer  the  following  questions:  
 
  a.  What  are  the  three  trends  that  have  taken  place  for  the  last  25  years?  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
 
 

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  b.  Identify  three  instances  mentioned  by  Enriquez  in  his  speech  related  to    
                             evolution.  
         ______________________________________________________________  
           ______________________________________________________________  
         ______________________________________________________________  
 
  c.  Do  you  believe  that  we  will  evolve  into  Homo  evolutis?  
                             ______________________________________________________________  
             ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
 
  d.  What  ideas  did  u  get  from  the  speech?  Discuss.  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
         ______________________________________________________________  
   
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

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