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NEWER ADVANCES IN GENETINEWER ADVANCES IN GENETI

BY,BY,

DR.M.L KULKARNIDR.M.L KULKARNI

DR.GAYATHRI.K DR.GAYATHRI.K

DAVANAGERE.DAVANAGERE.

REVIEWED BYREVIEWED BY

DR.SUBRAMANYA.N.K DR.SUBRAMANYA.N.K

BANGALOREBANGALORE

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DNA BASICSDNA BASICS

All genetic mte!il "DNA# c$ntine% in All genetic mte!il "DNA# c$ntine% in &ingle ge!m cell "&'e!m n% $()m# i& *n$+n&ingle ge!m cell "&'e!m n% $()m# i& *n$+n& )mn gen$me, tt c$ntin& ll& )mn gen$me, tt c$ntin& llin-$!mti$n $- li-e.in-$!mti$n $- li-e.

St!)ct)!eSt!)ct)!e

DNA e/i&t& & %$)0le &t!n%e% eli/.DNA e/i&t& & %$)0le &t!n%e% eli/.

Te n$l$g1 $- !$'e l%%e! cn 0e gi(en t$Te n$l$g1 $- !$'e l%%e! cn 0e gi(en t$te &t!)ct)!e $- DNA .Ec &i%e $- tete &t!)ct)!e $- DNA .Ec &i%e $- tel%%e! i& m%e )' $- cin& $- &)g!"&# n%l%%e! i& m%e )' $- cin& $- &)g!"&# n%

'$&'te&"2#.Te &te' $- l%%e! i& m%e'$&'te&"2#.Te &te' $- l%%e! i& m%e)' $- 3 nit!$gen$)& 0&e& -!$m 3 '$le&)' $- 3 nit!$gen$)& 0&e& -!$m 3 '$le&'!$4ecting in+!% n% 3 0&e& !e 4$ine% 01'!$4ecting in+!% n% 3 0&e& !e 4$ine% 011%!$gen 0$n%.1%!$gen 0$n%.

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Te nit!$gen$)& 0&e& c$n&i&t $- teTe nit!$gen$)& 0&e& c$n&i&t $- te')!ine&"%enine5A, g)nine5G#')!ine&"%enine5A, g)nine5G#

21!imi%ine&"c1t$&ine5C, t1mine5T#.21!imi%ine&"c1t$&ine5C, t1mine5T#.

A l+1& 'i!& +it T n% G +it C.A l+1& 'i!& +it T n% G +it C.

Ec 'i! $- c$m'lement!1Ec 'i! $- c$m'lement!1nit!$gen$)& 0&e i& te!me% 60&enit!$gen$)& 0&e i& te!me% 60&e

'i!7.'i!7.

A n)cle$ti%e i& %e8ne% & )nitA n)cle$ti%e i& %e8ne% & )nit

c$n&i&ting $- '$&'te, &)g! n%c$n&i&ting $- '$&'te, &)g! n%

nit!$gen$)& 'i!.nit!$gen$)& 'i!.

Te $!%e! $- 0&e $n $ne &ingle, &t!n%Te $!%e! $- 0&e $n $ne &ingle, &t!n%

i& *n$+n & DNA &e9)ence. Te DNA i&i& *n$+n & DNA &e9)ence. Te DNA i&M.L.K G.K

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M.L.K

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GENE STRUCTUREGENE STRUCTURE

Gene& !e &egment& $- DNA &t!etce&Gene& !e &egment& $- DNA &t!etce&

n% !e t!n&c!i0e% int$ RNA n% lte!n% !e t!n&c!i0e% int$ RNA n% lte!t!n&c!i0e% int$ '!$tein&.t!n&c!i0e% int$ '!$tein&.

Gene& !e c$m'$&e% $- e/$n& "teGene& !e c$m'$&e% $- e/$n& "te

c$%ing !egi$n # n% int!$n&"te n$nc$%ing !egi$n # n% int!$n&"te n$n

c$%ing !egi$n #.c$%ing !egi$n #.

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GENE STRUCTUREGENE STRUCTURE

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GENE ACTIONGENE ACTION

T!n&c!i'ti$nT!n&c!i'ti$n $ne &t!n% $- &t!)ct)!l$ne &t!n% $- &t!)ct)!l

gene DNA -$!m& tem'lte -$! tegene DNA -$!m& tem'lte -$! te

&1nte&i& $- RNA *n$+n & me&&enge!&1nte&i& $- RNA *n$+n & me&&enge!RNA"m RNA#.RNA"m RNA#.

T!n&lti$nT!n&lti$n A t!i'let $- 0&e& clle% A t!i'let $- 0&e& clle%

c$%$n c$%e& -$! &1nte&i& $- '!tic)l!c$%$n c$%e& -$! &1nte&i& $- '!tic)l!

min$ci%, mRNA 0ec$me& te tem'ltemin$ci%, mRNA 0ec$me& te tem'lte

in te !i0$&$me -$! t!n&lti$n.in te !i0$&$me -$! t!n&lti$n.M.L.K

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GENE ACTIONGENE ACTION

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RECOMBINANT DNA TECHNOLOGRECOMBINANT DNA TECHNOLOG

Te '!$ce&& $-Te '!$ce&& $-

mni')lting geneticmni')lting geneticmte!il, in +ic DNA i&mte!il, in +ic DNA i&

!ti8cill1 c)t, 4$ine% n%!ti8cill1 c)t, 4$ine% n%

&1nte&i:e% i& te!me% &&1nte&i:e% i& te!me% &DNA !ec$m0inntDNA !ec$m0innt

tecn$l$g1 $! Genetictecn$l$g1 $! Genetic M.L.K

G.K

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USES O; DNA RECOMBINANTUSES O; DNA RECOMBINANT

TECHNOLOGY TECHNOLOGY

i.Gene &t!)ct)!e<M''ing<;)ncti$n.i.Gene &t!)ct)!e<M''ing<;)ncti$n.ii. Clinicl Genetic&ii. Clinicl Genetic&

iii. .2!eim'lntti$n genetic %ign$&i&.iii. .2!eim'lntti$n genetic %ign$&i&. . 2!entl %ign$&i&.. 2!entl %ign$&i&. .2!e&1m't$mtic %ign$&i&..2!e&1m't$mtic %ign$&i&. . C!!ie! %etecti$n.. C!!ie! %etecti$n. iii. Dign$&i& n% 't$gene&i& $- %i&e&eiii. Dign$&i& n% 't$gene&i& $- %i&e&e .Genetic.Genetic .In-ecti(e.In-ecti(e . Mlignnt.. Mlignnt. i(. Bi$&1nte&i&i(. Bi$&1nte&i&

.Rec$m0innt '!$%)ct&.Rec$m0innt '!$%)ct& .Vccine&.Vccine& (. T!etment $- genetic %i&e&e&(. T!etment $- genetic %i&e&e& (i. Gene te!'1.(i. Gene te!'1.

M.L.K M.L.K

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DNA RECOMBINANT TECHNOLODNA RECOMBINANT TECHNOLO

M.L.K

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2OLYMERASE CHAIN REACTIO2OLYMERASE CHAIN REACTIO

2CR i& '$+e!-)l tecn$l$g1 tt i&2CR i& '$+e!-)l tecn$l$g1 tt i&

)&e% t$ m'li-1 te DNA &egment $-)&e% t$ m'li-1 te DNA &egment $-

inte!e&t i.e,=m$lec)l! '$t$c$'1ing=.inte!e&t i.e,=m$lec)l! '$t$c$'1ing=.

STE2SSTE2S

T!get DNA i& %ent)!e% 01 et n%T!get DNA i& %ent)!e% 01 et n%

%$)0le &tn%e% DNA i& &e'e!te% int$%$)0le &tn%e% DNA i& &e'e!te% int$

t+$ &ingle &t!n%&.t+$ &ingle &t!n%&.2!ime!& !e %%e% t$ DNA &t!n%&,2!ime!& !e %%e% t$ DNA &t!n%&,

te&e '!ime!& 10!i%i:e t$te&e '!ime!& 10!i%i:e t$

c$m'lement!1 -!gment& $- n$+c$m'lement!1 -!gment& $- n$+

&ingle &t!n%e% t!get DNA.&ingle &t!n%e% t!get DNA.M.L.K

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Ten 0&e& n% tg '$l1me!&e !eTen 0&e& n% tg '$l1me!&e !e

%%e%, +ile tem'e!t)!e i&%%e%, +ile tem'e!t)!e i&

inc!e&e%.inc!e&e%.

Te 0&e& !e )&e% & 0)il%ing 0l$c*Te 0&e& !e )&e% & 0)il%ing 0l$c*

-$! &1nte&i& $- DNA &t!n%& n%-$! &1nte&i& $- DNA &t!n%& n%'$l1me!&e 0egin& t$ &&em0le tem.'$l1me!&e 0egin& t$ &&em0le tem.

S)c c1cle& !e !e'ete% &e(e!lS)c c1cle& !e !e'ete% &e(e!l

time& )t$mticll1 n% in -e+ $)!&,time& )t$mticll1 n% in -e+ $)!&,

te t!get DNA &egment cn 0ete t!get DNA &egment cn 0em'li8e% t$ $ne milli$n -$l%.m'li8e% t$ $ne milli$n -$l%.

Te '!$%)ce% DNA cn 0e nl1&e%Te '!$%)ce% DNA cn 0e nl1&e%

)&ing l0elle% '!$0e&, &e9)encing $!)&ing l0elle% '!$0e&, &e9)encing $!%i!ecct (i&i$n +it U.V li t.

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A22LICATIONS O; 2CRA22LICATIONS O; 2CR

Genetic %i&$!%e! c)&e% 01 &ingle 0&e 'i!Genetic %i&$!%e! c)&e% 01 &ingle 0&e 'i!cnge&, &mll %eleti$n $! in&e!ti$n."Egcnge&, &mll %eleti$n $! in&e!ti$n."EgTl&&emi +it 0&e 'i! cnge, DMD +itTl&&emi +it 0&e 'i! cnge, DMD +itDNA %eleti$n.#DNA %eleti$n.#

Dign$&i& $- in-ecti$)& %i&e&e&.Dign$&i& $- in-ecti$)& %i&e&e&. Dign$&i& n% -$ll$+ )' $- cnce!&.Dign$&i& n% -$ll$+ )' $- cnce!&.

St)%1 $- 0$ne m!!$+ eng!-tment.St)%1 $- 0$ne m!!$+ eng!-tment.

DNA 8nge!'!inting -$! 'te!nit1 c&e& n%DNA 8nge!'!inting -$! 'te!nit1 c&e& n%

-$!en&ic ')!'$&e&.-$!en&ic ')!'$&e&. HLA t1'ing -$! 0$ne m!!$+ n% *i%ne1HLA t1'ing -$! 0$ne m!!$+ n% *i%ne1

t!n&'lntti$n.t!n&'lntti$n.

St)%1 $- gene lin*ge n% gene m''ing.St)%1 $- gene lin*ge n% gene m''ing.

St)%1 $- e($l)ti$n.St)%1 $- e($l)ti$n. M.L.K

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2OLYMERASE CHAIN REACTION2OLYMERASE CHAIN REACTION

TECHNI>UETECHNI>UE

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SOUTHERN BLOT HYBRIDI?ATISOUTHERN BLOT HYBRIDI?ATI

In ti& tecni9)e, 8!&t te DNA i& c)t in t$In ti& tecni9)e, 8!&t te DNA i& c)t in t$-!gment& 01 te )&e $- 0cte!il !e&t!icti$n-!gment& 01 te )&e $- 0cte!il !e&t!icti$nen:1me&, te&e 'iece& !e ten &)04ecte% t$en:1me&, te&e 'iece& !e ten &)04ecte% t$elect!$'$!e&i& in n g!$&e gel, &mlle!elect!$'$!e&i& in n g!$&e gel, &mlle!-!gment& m$(e -&te! tn l!ge! $ne&.-!gment& m$(e -&te! tn l!ge! $ne&.

Te g!$&e gel +it DNA t!c*& i&Te g!$&e gel +it DNA t!c*& i&t!n&-e!!e% -$! 'e!mnent !ec$!% 01t!n&-e!!e% -$! 'e!mnent !ec$!% 01'lcing nit!$5cell)l$&e ''e! $n t$' $- te'lcing nit!$5cell)l$&e ''e! $n t$' $- teg!$&e gel,!etining te lignment $-g!$&e gel,!etining te lignment $--!gment&"0l$tting#.-!gment&"0l$tting#.

Te !ec$!% i& ten +&e% in &$l)ti$nTe !ec$!% i& ten +&e% in &$l)ti$nc$ntining !%i$l0elle% DNA '!$0e +itc$ntining !%i$l0elle% DNA '!$0e +it'$&'$!$)& @3.Te '!$0e 8n%& it&'$&'$!$)& @3.Te '!$0e 8n%& it&c$m'lement!1 -!gment& $- DNA, )nite&c$m'lement!1 -!gment& $- DNA, )nite&+it it "H10!i%i:ti$n#.+it it "H10!i%i:ti$n#.

Ti& 10!i%i:e% -!gment cn 0e i%enti8e%Ti& 10!i%i:e% -!gment cn 0e i%enti8e%M.L.K G.K

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SOUTHERN BLOTSOUTHERN BLOT

HYBRIDI?ATIONHYBRIDI?ATION

M.L.K

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SOUTHERN BLOTSOUTHERN BLOT

HYBRIDI?ATIONHYBRIDI?ATION

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A22LICATIONSA22LICATIONS

T$ 8n% te '!e&ence $- T$ 8n% te '!e&ence $-

'!tic)l! gene $! '!t $- te'!tic)l! gene $! '!t $- te

-!gment in te &m'le DNA.-!gment in te &m'le DNA.T$ *n$+ +ete! te &i:e $- teT$ *n$+ +ete! te &i:e $- te

DNA -!gment c!!1ing te gene i&DNA -!gment c!!1ing te gene i&

n$!ml, inc!e&e% $! %ec!e&e%.n$!ml, inc!e&e% $! %ec!e&e%.

T$ i%enti-1 '!e&ence $- l!geT$ i%enti-1 '!e&ence $- l!ge

%eleti$n&, %)'licti$n& $! l!ge%eleti$n&, %)'licti$n& $! l!ge

gene !!ngement&.gene !!ngement&. M.L.K

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;LUOROSCENCE INSITU HYBRIDI?ATION";LUOROSCENCE INSITU HYBRIDI?ATION"

Te DNA $- te &)04ect i& 8!&tTe DNA $- te &)04ect i& 8!&t%ent)!e% 01 et int$ t+$ &e'!te%ent)!e% 01 et int$ t+$ &e'!te&t!n%&.&t!n%&.

Te )$!e&cet '!$0e $- inte!e&t i&Te )$!e&cet '!$0e $- inte!e&t i&

%%e% t$ te %ent)!e% DNA%%e% t$ te %ent)!e% DNA&m'le,+ic ten 10!i%i:e&")nite&#&m'le,+ic ten 10!i%i:e&")nite&#+it te c$m'lement!1 &egment i-+it te c$m'lement!1 &egment i-'!e&ent n% ten !e-$!m& int$ %$)0le'!e&ent n% ten !e-$!m& int$ %$)0le

eli/"!e nnel&#eli/"!e nnel&# Te !ec$m0innt DNA i& ten &eenTe !ec$m0innt DNA i& ten &een

t!$)g )$!e&centt!$)g )$!e&centmic!$&c$'e,+ic %etect& temic!$&c$'e,+ic %etect& te

'!e&cence $! 0&ence $- te'!e&cence $! 0&ence $- te

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DNA mic!$!!1 tecni9)eDNA mic!$!!1 tecni9)e

Ti& tecni9)e i& )&e% t$ &t)%1 te e/'!e&&i$n $-Ti& tecni9)e i& )&e% t$ &t)%1 te e/'!e&&i$n $-mn1 gene& t time n% tt t$$ $n &ingle &li%emn1 gene& t time n% tt t$$ $n &ingle &li%e*n$+n & DNA CHI2.*n$+n & DNA CHI2.

DNA Ci' i& gl&& &li%e in +ic &e(e!l minit)!eDNA Ci' i& gl&& &li%e in +ic &e(e!l minit)!e+ell& !e !!nge% in n $!%e! i.e mic!$!!1.+ell& !e !!nge% in n $!%e! i.e mic!$!!1.

In te&e $lig$n)cle$ti%e '!$0e& !e 'lce%.In te&e $lig$n)cle$ti%e '!$0e& !e 'lce%. Te m RNA i& e/t!cte% -!$m &m'le t$ 0e te&te%.Te m RNA i& e/t!cte% -!$m &m'le t$ 0e te&te%.

Ti& m RNA i& c$n(e!te% in t$ c DNA, +ic i& tenTi& m RNA i& c$n(e!te% in t$ c DNA, +ic i& ten

l0elle% +it )$!e&cent %1el0elle% +it )$!e&cent %1e.. Te m RNA -!$m n$!ml cell& i& l&$ e/t!cte% n%Te m RNA -!$m n$!ml cell& i& l&$ e/t!cte% n%

-!$m ti& c DNA !e ten mi/e% +it te-!$m ti& c DNA !e ten mi/e% +it te$lig$n)cle$ti%e '!$0e& tt !e 'lce% in +ell&$lig$n)cle$ti%e '!$0e& tt !e 'lce% in +ell&"mic!$ !!1#."mic!$ !!1#.

L&e! 0em& !e ten )&e% t$ e/cite teL&e! 0em& !e ten )&e% t$ e/cite te)$!$c!$me&. E/citing 'tte!n& %e'en% $n)$!$c!$me&. E/citing 'tte!n& %e'en% $n!elti(e m$)nt $- n$!ml $! 0n$!ml cDNA.!elti(e m$)nt $- n$!ml $! 0n$!ml cDNA.

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Anl1&i& i& %$ne 01 c't)!ing teAnl1&i& i& %$ne 01 c't)!ing te

%igitl imge n% lte! in nl1:ing in%igitl imge n% lte! in nl1:ing in

c$m')te!.c$m')te!.

Ti& i& gene e/'!e&&i$n &t)%1.Ti& i& gene e/'!e&&i$n &t)%1.";UNCTIONAL GENOMICS#.";UNCTIONAL GENOMICS#.

A22LICATIONSA22LICATIONSD!)g %e&igning, '!mc$genetic&,D!)g %e&igning, '!mc$genetic&,

cnce! !e&e!c, nl1:ing &e(e!lcnce! !e&e!c, nl1:ing &e(e!l

m)tti$n& in &ingle g$.m)tti$n& in &ingle g$.M.L.K G.K

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DNA MICROARRAYDNA MICROARRAY

TECHNI>UETECHNI>UE

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LINKAGE ANALYSISLINKAGE ANALYSIS

I- te %i&e&e c)&ing m)tti$n,cnn$tI- te %i&e&e c)&ing m)tti$n,cnn$t0e i%enti8e% $! %ic)lt t$ te&t, DNA0e i%enti8e% $! %ic)lt t$ te&t, DNA

m!*e!& &it)te% in $! ne! te genem!*e!& &it)te% in $! ne! te gene

cn 0e )&e% t$ t!ce te m)tte% genecn 0e )&e% t$ t!ce te m)tte% genein te -mil1.in te -mil1.

A22LICATIONSA22LICATIONS

2!entl %ign$&i&.2!entl %ign$&i&.

C!!ie! %etecti$n.C!!ie! %etecti$n.

D.M.DD.M.D

Hem$'iliHem$'iliM.L.K

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EN?YME RE2LACEMENT THERA2Y EN?YME RE2LACEMENT THERA2Y

Ti& te!'1 i& '$tentilTi& te!'1 i& '$tentil

t!etment -$! ce!tin genetict!etment -$! ce!tin genetic

%i&e&e&.Ti& met$% $- t!etment%i&e&e&.Ti& met$% $- t!etment

i& %e'en%ent $n te %eli(e!1 $- tei& %e'en%ent $n te %eli(e!1 $- teint!(en$)&l1 %eli(e!e% en:1me ,t$int!(en$)&l1 %eli(e!e% en:1me ,t$

te cell &)!-ce !ece't$! +e!e tete cell &)!-ce !ece't$! +e!e te

en:1me i& %e8cient.en:1me i& %e8cient.

Te 8!&t &)cce&&-)l te!'1 $-Te 8!&t &)cce&&-)l te!'1 $-

en:1me !e'lcement +& %$ne inen:1me !e'lcement +& %$ne in

G)ce!7& %i&e&e.G)ce!7& %i&e&e.

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G)ce!7& %i&e&eG)ce!7& %i&e&eImigl)ce!&eImigl)ce!&e

;0!1 %i&e&e;0!1 %i&e&e

Agl&i%&eAgl&i%&eM)c$'$l1&cc!i%$&i&M)c$'$l1&cc!i%$&i&

Al%)!:1meAl%)!:1me

M)c$'$l1&cc!i%$&i& (M)c$'$l1&cc!i%$&i& (Gl&)l-&eGl&)l-&e

M)c$'$l1&cc!i%$&i& M)c$'$l1&cc!i%$&i&

I%!&)l-&eI%!&)l-&e

DISEASE EN?YMEDISEASE EN?YME

RE2LACEDRE2LACED

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GENE THERA2Y GENE THERA2Y

Gene te!'1 +& c$nce't)li:e%Gene te!'1 +& c$nce't)li:e%

in e!l1 F& n% & &)04ecte%in e!l1 F& n% & &)04ecte%

t$ mn1 clinicl t!il& &ince ten.t$ mn1 clinicl t!il& &ince ten.

Gene te!'1 i& n$(el ''!$cGene te!'1 i& n$(el ''!$c

t$ t!et, c)!e, $! )ltimtel1t$ t!et, c)!e, $! )ltimtel1

'!e(ent %i&e&e01 cnging te'!e(ent %i&e&e01 cnging te

e/'!e&&i$n $- 'e!&$n7& gene&.e/'!e&&i$n $- 'e!&$n7& gene&.

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A22ROACHES TO GENEA22ROACHES TO GENE

THERA2Y THERA2Y

E/ (i($ ''!$cE/ (i($ ''!$c Te t!get cell& ! Te t!get cell& !

!em$(e% -!$m te 0$%1 n% ne+!em$(e% -!$m te 0$%1 n% ne+

gene i& int!$%)ce% in(it!$ 01 $ne $-gene i& int!$%)ce% in(it!$ 01 $ne $-tete

gene %eli(e!1 met$%&.gene %eli(e!1 met$%&.

In (i($ ''!$cIn (i($ ''!$c "Di!ect gene "Di!ect genet!n&-e!# Te te!)'etic gene i&t!n&-e!# Te te!)'etic gene i&

int!$%)ce% %i!ectl1 int$ te ecte%int!$%)ce% %i!ectl1 int$ te ecte%

ti&&)e +it$)t !em$(ing cell& -!$mti&&)e +it$)t !em$(ing cell& -!$mM.L.K

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TY2ES O; GENE THERA2Y TY2ES O; GENE THERA2Y

S$mtic cell te!'1S$mtic cell te!'1 ti& in($l(e& ti& in($l(e&in&e!ti$n $- te!)'etic gene int$in&e!ti$n $- te!)'etic gene int$

&$mtic cell& &)c &&$mtic cell& &)c &

80!$0l&t&,m1$0l&t&,e'itelil80!$0l&t&,m1$0l&t&,e'itelil

cell&,en%$telil cell&,ne!($)&,glilcell&,en%$telil cell&,ne!($)&,glilcell&,etc.cell&,etc.

Ge!mline te!'1Ge!mline te!'1 Ti& in($l(e& te Ti& in($l(e& te

int!$%)cti$n $- -$!eign gene int$ ge!mint!$%)cti$n $- -$!eign gene int$ ge!mcell& li*e &'e!m,$()m,$! -e!tili:e%cell& li*e &'e!m,$()m,$! -e!tili:e%

egg,!e&)lting in tei! e/'!e&&i$n in 0$tegg,!e&)lting in tei! e/'!e&&i$n in 0$t

&$mtic & +ell & ge!m cell& $- te&$mtic & +ell & ge!m cell& $- te

$&'!ing$&'!ing. M.L.K

G.K

7/23/2019 16 IAP


METHODS O; GENE DELIVERY METHODS O; GENE DELIVERY

21&icl21&icl

.21&icl in4ecti$n.21&icl in4ecti$n

.Mic!$ in4ecti$n.Mic!$ in4ecti$n.Ae!$&$l.Ae!$&$l

.Gene g)n.Gene g)n

CemiclCemicl

.Clci)m '$&'te.Clci)m '$&'te

.DEAE5%e/t!n.DEAE5%e/t!n

.Li'$&$me&.Li'$&$me&

Bi$l$giclBi$l$gicl

. Vi!l (ect$!&. Vi!l (ect$!&

. Ret!$ (i!)&. Ret!$ (i!)&

.A%en$ (i!)&.A%en$ (i!)&

.He!'e& &im'le/ (i!)&.He!'e& &im'le/ (i!)&

Ne$5$!gn im'lnt&Ne$5$!gn im'lnt& Ti&&)e t!n&'lntti$nTi&&)e t!n&'lntti$n

H)mn !ti8cil c!$m$&$meH)mn !ti8cil c!$m$&$me

Ote!&Ote!&

Rece't$! me%ite% %eli(e!1Rece't$! me%ite% %eli(e!1

Vi!ll1 %i!ecte% en:1me '!$%!)g te!'1.Vi!ll1 %i!ecte% en:1me '!$%!)g te!'1.

M.L.K

G.K

7/23/2019 16 IAP


GENE THERA2Y USING ADENOVIRUSGENE THERA2Y USING ADENOVIRUS

VECTORVECTOR

M.L.K

G.K

7/23/2019 16 IAP


C1&tic 80!$&i&C1&tic 80!$&i&Al'5 ntit!1'&in %e8cienc1Al'5 ntit!1'&in %e8cienc1

Hem$'ili A n% BHem$'ili A n% B

G)ce!7& %i&e&eG)ce!7& %i&e&eBet5em$gl$0in$'tie&Bet5em$gl$0in$'tie&

;milil 1'e!c$le&te!$lemi;milil 1'e!c$le&te!$lemi

2en1l*et$n)!i.2en1l*et$n)!i.

M.L.K

G.K

Following are the disorders under trial for geneFollowing are the disorders under trial for gene

therapy:therapy:

7/23/2019 16 IAP


Thank you

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