ALEJANDRA VARGAS VELÁSQUEZME D I C I N E S T U D E N TI I I S E ME S T E R              T e a c h e r , LINA MARÍA MARTÍNE...
DN  A RN   A                  Health                   I t c o mp l yPr ot            t h e c e n t r a l                 ...
SCI ENT I                    STS   CRE AT E NOV                    EL RNA          REPAI                    R      T E CHN...
S CI E NT I S T S CRE AT E NOV E L     RNA RE P AI R T E CHNOL OGY"F o r a l o n g t i m e i t wa s    t h o u g h t    t ...
S CI E NT I S T S CRE AT E NOV E L  RNA RE P AI R T E CHNOL OGY                                           Th e            ...
S CI E NT I S T S CRE AT E NOV E L   RNA RE P AI R T E CHNOL OGY             Th e Th e        t r i                       ...
S CI E NT I S T S CRE AT E NOV E L   RNA RE P AI R T E CHNOL OGYOBS E RV AT I ON Mo   l   e c u l a rg e   n   e t i c s  ...
SCI ENT I ST S CRE AT E P OT E NTMO L E C U L E S A I ME D   AT T REAT I NG     MU S C U L A R    DY S T ROP HY           ...
SCI ENT I ST S CREAT E        P O T E N T MO L E C U L E S A I ME D          A T T R E A T I N G MU S C U L A R           ...
SCI ENT I ST S CREAT EP O T E N T MO L E C U L E S A I ME D  A T T R E A T I N G MU S C U L A R           DY S T ROP HY   ...
SCI ENT I ST S CREAT E    P O T E N T MO L E C U L E S A I ME D      A T T R E A T I N G MU S C U L A R               DY S...
SCI ENT I ST S CREAT E P O T E N T MO L E C U L E S A I ME D   A T T R E A T I N G MU S C U L A R            DY S T ROP HY...
ME D I C A L   UT I L I T YMo l e c u l a r b i o l o g y i s   i n c r e a s i n g l y u s e d          i n c l i n i c a...
ME D I C A L                  UT I L I T YTh e         mo s t      p r o mi s i n g        f i e l d       o fa c t   i   ...
ME D I C A L           UT I L I T YI t i si mp o r t a n tt o n o t et h a t t op r e v e n tt h i s f r o mo c c u r r i ...
ME D I C A L                 UT I L I T YBecause the molecular biology isrelated to the areaof ​pathology, t h emo l e c u...
BI BL I OGRAP H        Y    • MARTINEZ SÁNCHEZ, Lina María.      Biología molecular. 2. ed. Medellín:      U P B . F a c ....
T HEEND
Scientists Create Novel RNA Reapir Techonology and Scientists Ceate Potent Molecules Aimed at Treating Muscular Dystrophy
Scientists Create Novel RNA Reapir Techonology and Scientists Ceate Potent Molecules Aimed at Treating Muscular Dystrophy
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Scientists Create Novel RNA Reapir Techonology and Scientists Ceate Potent Molecules Aimed at Treating Muscular Dystrophy

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Scientists Create Novel RNA Reapir Techonology and Scientists Ceate Potent Molecules Aimed at Treating Muscular Dystrophy

  1. 1. ALEJANDRA VARGAS VELÁSQUEZME D I C I N E S T U D E N TI I I S E ME S T E R T e a c h e r , LINA MARÍA MARTÍNEZ SÁNCHEZ B I O L OGY MO L E C U L A R F E B R A U R Y 27, 2012 MEDELLÍN, C O L O M B I A
  2. 2. DN A RN A Health I t c o mp l yPr ot t h e c e n t r a l d o g ma .e i ns DNDISEASE A Al t e r e d RN t h e c e n t r a l A d o g ma . Pr ot e i ns
  3. 3. SCI ENT I STS CRE AT E NOV EL RNA REPAI R T E CHNOL OGYSc i e n t i s t sf r o m t h eFl o r i d ac a mp u s o f T h eSc r i p p sRe s e a r c hI n s t i t u t ea r e a l r e a d yh a r d a t wo r kt o e x t e n d t h e “E x p a n d e dl a b s s o f Re s u l t t r i p l e tf i n d i n n vse s t i g r e pe a t “a b o u t i t g h te i o a Th r e eR N A .e c nt i De f v e RNA n u c l e o t i d e s t h a t Ca u r e p e a t e d s e s ma n y mo r e t i me s t h a n
  4. 4. S CI E NT I S T S CRE AT E NOV E L RNA RE P AI R T E CHNOL OGY"F o r a l o n g t i m e i t wa s t h o u g h t t h a t o n l y t h e p r o t e i n t r a n s l a t e d f r o m t h i s t y p e o f RNA wa s t o x i c , b u t i t h a s b e e n s h o wn r e c e n t l y t h a t b o t h t h e p r o t e i n a n d t h e RNA a r e t o x i c . Ou r d i s c o v e r y o f a s ma l l mo l e c u l e t h a t b i n d s t o RNA a n d s h u t s o f f i t s t o x i c i t y n o t o n l y f u r t h e r d e mo n s t r a t e s t h a t t h e RNA i s
  5. 5. S CI E NT I S T S CRE AT E NOV E L RNA RE P AI R T E CHNOL OGY Th e e a r l y - I N T HE s t a g e RESEARCH a b n o r ma l i t i e s Sc i e I mp r o v e m n t i s e n t t s Us e d 4, 6- To x i c d i a mi d CAG i n o -2- t r i p l p h e n y l e t i n d o l e r e p e a t o (D A P I ) i n h i b t i t a
  6. 6. S CI E NT I S T S CRE AT E NOV E L RNA RE P AI R T E CHNOL OGY Th e Th e t r i Th e r e a r e a t r i p l e Th e n u mb e r o f p l e t t r i p r e p l e t n e u r o l o g i c a l t r e p e r e p e a t a t a n d Th e e a t t r i p l e t r e p e a t n e u r o mu s c u l a r i n h e r i t e d d i s e a s e s wh o s e The mo l e c u l a r t r i pl eDe f e c t b a s i s i s t h e t i v e r e pe a t a l t e r a t i o n i n t h e RNA, t h a t RNA h a s r e p e a t e d n u c l e o t i d e N o r ma l u n i t s , u s u a l l y RNA i n t r i p l e t s . Th e n o r ma l
  7. 7. S CI E NT I S T S CRE AT E NOV E L RNA RE P AI R T E CHNOL OGYOBS E RV AT I ON Mo l e c u l a rg e n e t i c s h a sf a c i l i t a t ed r a ma t i c a l l y t ot h e p o s s i b i l i t i e so f g e n e t i c O n e o f t h e ma i nma n i p u l a t i o n o f o b j e c t i v e s o f t h et h e e l e me n t s o f r e s e a r c h i s t oi n t e r e s t , s u c h a s c u r e , r e d u c e s o med i s e a s e s , t h a n k s t o a l t e r a t i o n s , d e c r e a s et h i s , i t i s t h e e f f e c t c a u s e d b yp o s s i b l e g e t t h e d i s e a s e o r s i mp l yi n f o r ma t i o n , k n o w l
  8. 8. SCI ENT I ST S CRE AT E P OT E NTMO L E C U L E S A I ME D AT T REAT I NG MU S C U L A R DY S T ROP HY Ac t a g a i n s t Mo l e RNA c u l e d e f e c t s De s i St u d y g n e d i n g
  9. 9. SCI ENT I ST S CREAT E P O T E N T MO L E C U L E S A I ME D A T T R E A T I N G MU S C U L A R DY S T ROP HYS o me d e f e c t s o f R N A a r e d i r e c t l y r e s p o n s i b l ef o r ma n y d i s e a s e s , o n e e x a mp l e i s t h e mo s tc o mmo n f o r m o f mu s c u l a r d y s t r o p h y i na d u l t s , m y o t o n i c d y s t r o p h y t y p e 1. Ma n y r e s e a r c h h a s s h o wn t h a t n e w c o mp o u n d s i mp r o v e d a n u mb e r o f b i o l o g i c a l d e f e c t s a s s o c i a t e d w i t h my o t o n i c d y s t r o p h y t y p e 1 i n c e l l c u l t u r e a n d a n i ma l mo d e l s . F o r t h i s r e a s o n , RNA i s n o w u s e d a s a t a r g e t p o i n t f o r s o me s p e c i f i c d i s e a s e s .
  10. 10. SCI ENT I ST S CREAT EP O T E N T MO L E C U L E S A I ME D A T T R E A T I N G MU S C U L A R DY S T ROP HY Bi n d i n g t o aMy o t o n i c By p a r t i c u ld y s t r o p h Di s e a s e a r y t y p e 1 p r o t e i n , M I n v o l v L eh d s t a e Bo iL 1 D N e s n g r e p e t i t i RNA o n o f I n a c t i v e d e f e c t CUG i n A s eR Ne s o f r i A Kn o wn I n t h i s a s a s e tq hur ee n c e c a s e e Me a n u c l e o t i d e"t r i p l e t n s s r e p e a t e d r e p e a t ” i n a n i n d i v i d u a l s g e n e t i c
  11. 11. SCI ENT I ST S CREAT E P O T E N T MO L E C U L E S A I ME D A T T R E A T I N G MU S C U L A R DY S T ROP HYDe f e My o t o n i cc t i v d y s t r o p h Me RNA y o t o n i c Sy mp t o ms d y s t r o p h y o f t h i s t y p e 1 y v a r i a b l e t y p e 1 d i s e a s eDe f e c c a n t i v e i n c l u d e wa s t i n g o fp r o t e t h e i n s mu s c l e s
  12. 12. SCI ENT I ST S CREAT E P O T E N T MO L E C U L E S A I ME D A T T R E A T I N G MU S C U L A R DY S T ROP HYOBS E RV AT I ON"T h e r e a r e l i mi t l e s s RNA t a r g e t s i n v o l v e d i n d i s e a s e ; t h e q u e s t i o n i s h o w t o f i n d s ma l l mo l e c u l e s t h a t b i n d t o t h e m , We v e a n s we r e d t h a t q u e s t i o n b y r a t i o n a l l y d e s i g n i n g t h e s e c o mp o u n d s t h a t t a r g e t t h i s R N A . T h e r e s n o r e a s o n t h a t o t h e r T h ro o u g h v s t su m ai l e l s ,m o l t e cw al se s t a r g e t i n b i a c t i e d i u g p o s s ir b l R N A s t o c o ui l d de nn tt i f bye o t h e e d e v e l o p e d c e ri tn a i n a mso i l m i c l u a re s a p p ra o a c h .” D i s n e u s g e l t h t y h a v e . S c ih e n c ea D a il li y y(F e b ro a u r y 22, 2012). s a i d t e b i t t r e d u c e t h e d e f e c t s o f RNA, wh i c h i n t h i s c a s e c a u s e a t y p e o f mu s c u l a r a t r o p h y . I t mu s t e mp h a s i z e t h a t t h a n k s t h i s
  13. 13. ME D I C A L UT I L I T YMo l e c u l a r b i o l o g y i s i n c r e a s i n g l y u s e d i n c l i n i c a l d i a g n o s i s . A g r o wi n g n u mb e r o f d i s e a s e s c a n b e d e t e c t e d a n d mo n i t o r e d i n r e a l t i me , g e n e r a t i n g r e l e v a n t i n f o r ma t i o n f o r b e t t e r me d i c a l ma n a g e me n t o f p a t i e n t s .
  14. 14. ME D I C A L UT I L I T YTh e mo s t p r o mi s i n g f i e l d o fa c t i o n i s mo l e c u l a r b i o l o g y .Mu s t s e a r c h i n i n f o r ma t i o n t h ec a u s e o f t h e d i s e a s e , t h a t i t i sf o u n d i n t h e g e n e , a t y p e o fi n f o f o e , r m a t i c T h e p re o g rr a m t a l w h i o fh r k ce x p l a i n s t h d it s e a s e c e l i l n h a m a n y a t h e s t oma k e . a n d h o wc a s e s n o t i n a l l , i s t h e s a me a s t h a t s a y wh i c h t h e i n h e r i t a n c e o f t h e p a r e n t s i s a l t e r e d a n d o r d e r e d a wr o n g w a y t o d e v e l o p me n t . Ac c o r d i n g l y , ma n y g e n e s h a v e b e e n
  15. 15. ME D I C A L UT I L I T YI t i si mp o r t a n tt o n o t et h a t t op r e v e n tt h i s f r o mo c c u r r i n gt h e DNAa n d RNA My o t o n i c mu s c u l a rmu s t b e i n d y s t r o p h y (D M 1) i s t h e i n h e r i t e dp e r f e c t n e u r o mu s c u l a rc o n d i t i o n . d i s e a s e mo s t c o mmo n i n a d u l t s
  16. 16. ME D I C A L UT I L I T YBecause the molecular biology isrelated to the areaof ​pathology, t h emo l e c u l a rb i o l o g yl a b o r a t o r y u s e dd i a g n o s t i ct e c h n o l o g i e sa n d r e s e a r c h t oi d e n t i f yg e n e t i cmu t a t i o n s , l a b o ra t o r yc o n f i r ma t i o na n d c o n t r o l o f
  17. 17. BI BL I OGRAP H Y • MARTINEZ SÁNCHEZ, Lina María. Biología molecular. 2. ed. Medellín: U P B . F a c . de Medicina, 2006. 208 p . • SCRI P PS RESEARCH I NST I T UT E. Sc i e n t i s t s Cr e a t e No v e l RNA Re p a i r Te c h n o l o g y . Sc i e n c e Da i l y ( 17 J a n 2012). • J UPI T ER, F L . Sc i e n t i s t s Cr e a t e Po t e n t Mo l e c u l e s A i me d a t Tr e a t i n g Mu s c u l a r Dy s t r o p h y . Sc i e n c e Da i l y (
  18. 18. T HEEND

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