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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.html7/24/2019 Avneet -Gene Therapy
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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_DNA7/24/2019 Avneet -Gene Therapy
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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/Macromolecule7/24/2019 Avneet -Gene Therapy
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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.
02
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