Avneet -Gene Therapy

7/24/2019 Avneet -Gene Therapy

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GENE

THERAPY

By- AVNEET KAUR

.

MODERATOR- Prof .VIRENDRA SINGH

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What is gene therapy ?

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Imagine that you accidentally brokeone of your neighbor's windows

I. Stay silent: no one will ever find out

that you are guilty, but the window

doesn't get fixed.

II. Repair it with some tape: not the best

longterm solution.

III. !ut in a new window: not only do yousolve the problem, but also you do

the honorable thing.

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"any medi#al #onditions result from

flaws in one or more genes. What are

your treatment options?I. Stay silent: ignore the geneti# disorder

and nothing gets fixed.

II. $ry to treat the disorder with drugs orother approa#hes: depending on thedisorder, treatment may or may not be agood longterm solution.

III. !ut in a normal, fun#tioning #opy of thegene: if you #an do this, it may solve the

problem%E

N

E

T

H

E

R

A

P

Y

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What is Gene Therapy?

&ene therapyis !" i#s"rio# of $"#"s i#o %#

i#&i'i&(%)*s +"))s %#& iss("s o r"% %

&is"%s", s(+! %s % !"r"&i%ry &is"%s" i# !i+!

% &")""rio(s (%# %))")" is r"/)%+"& i! %

f(#+io#%) o#". T!" "+!#o)o$y is si)) i# is i#f%#+y

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(), *hromosomes, +&enes: ) rief Introdu#tion


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WHAT IS A C LL?

$asic building blocks of all li%ing things&

Human body composed of trillions of

cells&

"ell contain bodys hereditary material&

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NUCL US

Cells command center

Houses DNA

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E%+! +!rooso"

is %&" (/ of DNA

i$!)y +oi)"& %#y

i"s %ro(#&/ro"i#s +%))"&

!iso#"s !%

s(//or is sr(+(r".

What is a

chromosome?

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What is a gene?

G"#"s %r" %&" (/ of DNA.

E%+! +!rooso" +o#%i#s

%#y $"#"s.

I# !(%#s, $"#"s si8"- fro %

f" !(#&r"& DNA %s"s oor" !%# 2 i))io# %s"s.

H(%# !%s "si%"& """#

27,777 %#& 20,777 $"#"s13


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Ho Genes Wor!"""?

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Ho #o genes #irect the $ro#uction o% $roteins?

$"#"s +o#%i# !" i#for%io# #""&"& o %9"

f(#+io#%) o)"+()"s +%))"& /ro"i#s.

:o(r#"y fro $"#" o /ro"i# +o#siss of o %:or

s"/s; r%#s+ri/io# %#& r%#s)%io#.TRANSCRIPTI&N

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Why Genetic (isorders?

%)"r"& so !% !"

"#+o&"& /ro"i#s%r" (#%)" o +%rry

o( !"ir #or%)

f(#+io#s, $"#"i+&isor&"rs +%# r"s().

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Gene therapy -Background

1990 !he first gene therapy ournal

published, Human Gene Therapy

1990 !he first approved gene therapy

clinical trial too# place when $shanthi

%e&ilva, a ' year old girl (Maryland )

with $%$deficient &evere *ombined

+mmunodeficiency , was given her own !

cells engineered with a retroviral vector

carrying a normal $%$ gene.

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)$ubble $*+,

2333 -T!" firs $"#"

!"r%/y +(r" %s

r"/or"& !"# A)%i#

=is+!"r >P%riss(++""&"& i# o%))y

+orr"+i#$ +!i)&r"# i!

SID or @()" oy

sy#&ro"

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How It Works ?A '"+or &")i'"rs !" !"r%/"(i+ $"#" i#o %

/%i"#s %r$" +"))

T!" %r$" +"))s "+o" i#f"+"& i! !" 'ir%)

'"+or

T!" '"+ors $"#"i+ %"ri%) is i#s"r"& i#o !"

%r$" +"))

=(#+io#%) /ro"i#s %r" +r"%"& fro !" !"r%/"(i+

$"#" +%(si#$ !" +")) o r"(r# o % #or%) s%"

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-teps in Gene


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-teps in GeneTherapy

!/;CC"#+%r%.s#.+oC"&i%410126CG"#"T!"r%/y.!) 22
http://encarta.msn.com/media_461561269/Gene_Therapy.htmlhttp://encarta.msn.com/media_461561269/Gene_Therapy.html


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-omatic and Germ #ineGene Therapy

G"#" !"r%/y +%# %r$" so%i+ >o&y or

$"r >"$$ %#& s/"r +"))s.

I# so%i+ $"#" !"r%/y !" r"+i/i"#*s$"#o" is +!%#$"&, ( !" +!%#$" is #o

/%ss"& o# o !" #" $"#"r%io#. G"r )i#" $"#" !"r%/y !" #")y

i#ro&(+"& $"#" is /%ss"& o# o !"

offs/ri#$.

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TRANSFECTION

is the introduction of

DNA into animal orplant cells

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) new gene is in5e#ted into an adenovirus ve#tor, whi#h is

used to introdu#e the modified () into a human #ell. If the

treatment is su##essful, the new gene will ma6e a fun#tionalprotein. 25


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#imitation of (irect GeneInduction

Si/)"s "!o&

Fii"& %//ro%+!-

(s"& o#)y i! +"r%i#iss("s %#& r"(ir"s

)%r$" %o(#s of

DNA.

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!on %iral


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!on0%iralmethods

In5e#tion of (a6ed ()

!hysi#al "ethods :

1le#troporation

&ene &un

Sonoporation "agnetofe#tion

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In1ection of !aked


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In1ection of !aked(!/

si/)"s "!o& of #o#-'ir%) r%#sf"+io#.

)i#i+%) ri%)s +%rri"& o( of i#r%(s+()%r i#:"+io#

of % #%9"& DNA/)%si& o++(rr"& i! so"

s(++"ss.

I# %&&iio# ri%)s i! #%9"& PR/ro&(+,

s!o"& sii)%r or $r"%"r s(++"ss.

"))()%r (/%9" of #%9"& DNA is $"#"r%))y

i#"ffi+i"#.32
http://en.wikipedia.org/wiki/Naked_DNAhttp://en.wikipedia.org/wiki/Polymerase_chain_reactionhttp://en.wikipedia.org/wiki/Polymerase_chain_reactionhttp://en.wikipedia.org/wiki/Naked_DNA


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Physical 2ethods 3

1le#troporation:

Us"s s!or /()s"s of !i$!

'o)%$" o +%rry DNA

%+ross !" +")) "r%#".

s!o+9 +%(s"s "/or%ry

for%io# of /or"s i# !"+")) "r%#", %))oi#$

DNA o)"+()"s o /%ss

!ro($!.

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Effi+i"# ( !i$! r%" of +")) &"%! )ii"& is

(s", i#+)(&i#$ +)i#i+%) %//)i+%io#s.

171*$R8()4)7)(*-1 $R)(S91*$I8(

#" "!o& of ")"+ro/or%io#

By (si#$ % !i$!-'o)%$" /)%s% &is+!%r$", DNA

%s "ffi+i"#)y &")i'"r"& fo))oi#$ '"ry s!or

>i+ros"+o#& /()s"s.

Effi+i"# %#& +%(s"s )"ss +"))()%r &%%$".

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-onoporation

)ppli#ation 8f

ltrasound that +r"%"s

r%#si"# /or"s i# % +"))

"r%#", %))oi#$ &r($

o)"+()"s, /ro"i#s, or

for"i$# DNA o "#"r !"

+")).

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"hemical 2ethods3

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( d i


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(endrimers !i$!)y r%#+!"& %+roo)"+()"i! % s/!"ri+%)

s!%/".

*ationi# dendrimer > o#" i! % /osii'" s(rf%+"

+!%r$" is +o#sr(+"&.

-8W I$ W8R;S

I# /r"s"#+" of $"#"i+ %"ri%) s(+! %s DNA or

RNA i &o "/or%ry %sso+i%io# i! !" #(+)"i+

%+i&.O# r"%+!i#$ is &"si#%io# !" &"#&ri"r-#(+)"i+

%+i& +o/)" is !"# %9"# i#o !" +")) 'i%

"#&o+yosis47
http://en.wikipedia.org/wiki/Macromoleculehttp://en.wikipedia.org/wiki/Macromolecule


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1.- vivo gene therapy

.+n vivo gene therapy

/. /ntisense therapy

$

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/ntisense therapy

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P bl i h G


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Problems with GeneTherapy S!or Fi'"&

H%r& o r%/i&)y i#"$r%" !"r%/"(i+ DNA i#o$"#o" %#& r%/i&)y &i'i&i#$ #%(r" of +"))s/r"'"# $"#" !"r%/y for )o#$ i"


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Current tatus

=DA !%s# %//ro'"& %#y !(%# $"#" !"r%/y/ro&(+ for s%)"

R1)S8(S: I# 1666, 15-y"%r-o)& J"ss" G")si#$"r &i"& fro

()i/)" or$%# f%i)(r" 4 &%ys %f"r r"%"# foror#i!i#" r%#s+%roy)%s" &"fi+i"#+y.D"%! %s ri$$"r"& y s"'"r" i(#" r"s/o#s" o

%&"#o'ir(s +%rri"r

J%#(%ry 2773, !%) o (si#$ r"ro'ir(s '"+ors i#)oo& s" +"))s "+%(s" +!i)&r"# &"'")o/"&)"(9"i%-)i9" +o#&iio# %f"r s(++"ssf() r"%"# for-)i#9"& s"'"r" +oi#"& i(#o&"fi+i"#+y

&is"%s" 46


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nresolve! "#estions

Can gene expression $e controlle! in thepatient%

&hat happens i' nor(al gene isoverexpresse!%

)o* long *ill the therapy last%

&hat is the $est vector to #se%

&hat is the (ini(#( n#($er o' cells

nee!e! to in'ect to achieve s#ccess% 01


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!eferences

1.TETBOOK O= MIROBIOFOGL BL

ANANTNARALAN.

2.TETBOOK O= BIOHEMISTRL BL

SATLANARALAN.3.TETBOOK O= BIOHEMISTRL BL

HARPER.

4.GENETHERAPL HANDBOOK.

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Avneet -Gene Therapy