iPS  cells:  A  BREAKTHROUGH  IN  STEM  CELL  RESEARCH

Presented  by  Mridula  Ray

H+  2010

Outline

• Embryonic  Stem  Cells

• iPS  cells  –  turning  Adult  Cells  into  Embryonic-­‐like  cells

• ImplicaCons  in  research  &  drug  design

• ImplicaCons  in  medicine

Embryonic  Stem  Cells

• Self-­‐renewal   Can  divide  indefinitely  into  two  iden/cal  “daughter  cells”  that  have  the  same  

proper/es  as  the  original  cell

• Pluripotency   Can  turn  into  ANY  cell  in  the  body

Where  do  ES  cells  come  from?

Modified  fromh@p://www.biology-­‐online.com

What  can  ES  cells  do?

Courtesy  Biobusiness.tv

differen

CaCo

n

reprogramming

Induced  Pluripotent  Stem  (iPS)  Cells

• RevoluConary  technique  to  turn  cells  from  adult  Cssue  into  an  ES  cell-­‐like  state  

  Shinya  Yamanaka  2006

• RelaCvely  easy  to  do,  phenomenon  reproduced  by  many  labs  around  the  world

How  are  iPS  cells  made?

• Put  in  4*  genes  involved  in  development  and  cancer

• Conferred  ES  cell  like  properCes  

THE  TECHNIQUE

THE  EXPERIMENT

Replace  most  of  a  virus’s  DNA  with  human  DNA.The  part  of  the  virus  DNA  that  makes  it  dangerous  is  removed

Infect  a  human  cell The  the  human  DNA  (and  the  virus’s  DNA)  get  integrated  into  the  cell’s  DNA

DNAhuman  DNA

Embryonic  Stem  Cells

• Self-­‐renewal   Can  divide  indefinitely  into  two  iden/cal  “daughter  cells”  that  have  the  same  

proper/es  as  the  original  cell

• Pluripotency   Can  turn  into  ANY  cell  in  the  body

And  iPS  

Cells!!

WHAT  CAN  IPS  CELLS  DO?

The  near-­‐future  of  scienPfic  discovery

• Cannot  always  obtain,  idenCfy  or  purify  stem  cells  from  a  diseased  paCent

• Cannot  always  study  diseased  cells  in  a  lab  (limited  lifespan  even  if  you  can)

• May  take  years  to  see  effect

• Disease  model  is  improved• Drug  targets  found   discovery  improved• Toxicity  studies  are  improved

THE  PROBLEM

iPS  SOLUTION

iPS  cells  are  a  LONG  way  off  from  

• Mice  grown  from  iPS  cells  are  more  likely  to  get  cancer  than  natural  babies

• Is  an  iPS  cell  completely  reprogrammed  on  the  molecular  level?    

• Quality  standards?• Making  iPS  cells  takes  a  long  Cme!

• Stem  cells   increases  risk  of  cancer• Delivery  of  a  stem  cell  to  the  right  place  may  be  a  challenge!• Ge^ngthe  right  signals  to  the  stem  cells  is  hard  to  control

Autologous  versus  Allogenic

• Autologous  –  from  your  own  body• Allogenic  –  from  a  donor

Your  immune  system  can  recognize  “self”  from  “non-­‐self”

If  the  donor  does  not  have  similar  enough  geneCc  properCes  as  you,  your  immune  system  will  reject  the  transplanted  organ,  blood,  cells  etc

PaCents  who  have  had  organ  transplants  oden  have  to  take  immuno-­‐suppressive  medicines  for  the  rest  of  their  lives

Current  and  PotenPal  Uses  of  Stem  Cells

Current  Use• IVF• Bone  marrow  transplants• Cord  blood  banking• Drug  Screening  &  toxicity• Disease  models• Diabetes• Cornea  repair  (Australia,  India)• Skin  grads• Spinal  Cord  injury  (1st  ES  C  FDA  

approved    clinical  trial  in  the  US)  • Cardiac  and  bone/carClage  regen.• Full  organ  regeneraCon  challenges

PotenCal  use  with  iPS• N/A

• Autologous

• Not  necessary

• Personalized  medicine

• From  actual  diseased  cells

• Autologous

• Autologous  (no  difference)

• Autologous

• Autologous

THANK  YOU!