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Gopi Krishna Giri
 It is otherwise known as ‘Gene Pharming’.
 ‘Pharming’- Combination of Farming and
Pharmaceuticals.
 It refers to use of genetic engineering to insert a gene in
host animals to produce drugs or other beneficial products to
humans.
 It is used in various transgenic animals like,
1.Transgenic Mice
2.Transgenic Cattle
3.Transgenic Goat
4.Transgenic Pigs
5.Mosquitoes
 Production of pharmaceutically valuable human proteins
from milk of transgenic animals –”Animal Pharming”.
1.Super Mouse:
 Ralph Brinster (1982) develop first transgenic mouse – Super
Mouse.
 It used for Meat production.
 Introducing Rat Growth Hormone Gene in mouse
metalothionein promoter into fertilized eggs.
It has high level of growth hormone .
Attains 44 grams weight in 10 weeks compared to normal
sibiling are 29 grams.
2.Knockout Mice:
 Transgenic mice carries Knockout Gene (Non- Functional
gene) at place of gene of interest –Knockout Mice.
Introduction of Collagen Gene in mice genome produce
knockout mice suffering Osteogenesis.
It is used to treat human diseases Osteogenesis.
Another , introduction of HIV tat Gene in mice generate
knockout mice with Kaposi’s Sarcoma.
It is used to treat Kaposi’s Sarcoma diseases in human.
Transgenic mice are used as human disease models for AIDS,
Diabetics, Cancer,etc.,
Microinjection method used to create transgenic cattle with
novel gene.
Transgenic cows with K-Casein Gene insert ,produce milk rich
in K-Casein. This milk used for cheese making.
Transgenic Cattle with Lactose Gene insert ,produce milk
which is poor in lactose. This milk good for lactose intolerant
people.
It is used for Animal Pharming.
Transgenic Goats with tPA Gene tissue plasminogen
activator in milk.
tPA is used to dissolve Blood Clots.
Gene for Factor VIII, introduced into zygote of pig and it was
implanted in uterus of sow.
Sow produce transgenic pigs that could produce Factor VIII in
milk .
It is used to treat Haemophilia.
Transgenic Pigs with Human β- globulin gene , produce human
hemoglobin in their blood.
Gene codes for endotoxin of Plasmodium vivax,
introduced into zygote of mosquitoes.
It produced toxin of Malarial parasites in their Saliva. When it
suck blood ,they inject toxin into human blood.
Human immune system produce Ab against toxin so body resist
to Malarial parasites.
It isused to vaccinate against malaria, viral and bacterial
diseases in human.
Molecular Screens to find New Candidate Drugs
Target driven drug discovey have changed way to perform large
scale screening.
Target enables to generate materials cheap, flexible ,scaleable
and reliable.
To reduce risk of undesired compound effects.Develop
compound selectively for desired target over protein sharing.
Advance in bioinformatics, enables use to identify target related
sequence, development of parallel screening tools and assays.
 compound can be assesd in assays or 3-D structure .It will helps
to optimize drug design and used to compound screening such
as,In Silico Screening .
In Silico Screening:
Screening of ligands for matches to lead binding site of target –
Virtual Screening or In Silico Screening.
It can be done with programmed computer.
Computer based matching of binding of target with active site
of active lead – In Silico Daking.
Virutal screening start from 3D structure of target molecules by
X-ray crystallography or NMR spectroscopy.
3D structure obtained from Protein Data Bank.
Autodock program helps to predict interaction between target
and ligand with refer to geometric position of reactive groups,
bond angles,bond length, side chain flexibility and molecular
force fields.
Application of transgenic animals

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Application of transgenic animals

  • 2.  It is otherwise known as ‘Gene Pharming’.  ‘Pharming’- Combination of Farming and Pharmaceuticals.  It refers to use of genetic engineering to insert a gene in host animals to produce drugs or other beneficial products to humans.
  • 3.  It is used in various transgenic animals like, 1.Transgenic Mice 2.Transgenic Cattle 3.Transgenic Goat 4.Transgenic Pigs 5.Mosquitoes  Production of pharmaceutically valuable human proteins from milk of transgenic animals –”Animal Pharming”.
  • 4. 1.Super Mouse:  Ralph Brinster (1982) develop first transgenic mouse – Super Mouse.  It used for Meat production.  Introducing Rat Growth Hormone Gene in mouse metalothionein promoter into fertilized eggs. It has high level of growth hormone . Attains 44 grams weight in 10 weeks compared to normal sibiling are 29 grams.
  • 5. 2.Knockout Mice:  Transgenic mice carries Knockout Gene (Non- Functional gene) at place of gene of interest –Knockout Mice. Introduction of Collagen Gene in mice genome produce knockout mice suffering Osteogenesis. It is used to treat human diseases Osteogenesis. Another , introduction of HIV tat Gene in mice generate knockout mice with Kaposi’s Sarcoma. It is used to treat Kaposi’s Sarcoma diseases in human. Transgenic mice are used as human disease models for AIDS, Diabetics, Cancer,etc.,
  • 6. Microinjection method used to create transgenic cattle with novel gene. Transgenic cows with K-Casein Gene insert ,produce milk rich in K-Casein. This milk used for cheese making. Transgenic Cattle with Lactose Gene insert ,produce milk which is poor in lactose. This milk good for lactose intolerant people.
  • 7. It is used for Animal Pharming. Transgenic Goats with tPA Gene tissue plasminogen activator in milk. tPA is used to dissolve Blood Clots.
  • 8. Gene for Factor VIII, introduced into zygote of pig and it was implanted in uterus of sow. Sow produce transgenic pigs that could produce Factor VIII in milk . It is used to treat Haemophilia. Transgenic Pigs with Human β- globulin gene , produce human hemoglobin in their blood.
  • 9. Gene codes for endotoxin of Plasmodium vivax, introduced into zygote of mosquitoes. It produced toxin of Malarial parasites in their Saliva. When it suck blood ,they inject toxin into human blood. Human immune system produce Ab against toxin so body resist to Malarial parasites. It isused to vaccinate against malaria, viral and bacterial diseases in human.
  • 10. Molecular Screens to find New Candidate Drugs Target driven drug discovey have changed way to perform large scale screening. Target enables to generate materials cheap, flexible ,scaleable and reliable. To reduce risk of undesired compound effects.Develop compound selectively for desired target over protein sharing.
  • 11. Advance in bioinformatics, enables use to identify target related sequence, development of parallel screening tools and assays.  compound can be assesd in assays or 3-D structure .It will helps to optimize drug design and used to compound screening such as,In Silico Screening . In Silico Screening: Screening of ligands for matches to lead binding site of target – Virtual Screening or In Silico Screening. It can be done with programmed computer.
  • 12. Computer based matching of binding of target with active site of active lead – In Silico Daking. Virutal screening start from 3D structure of target molecules by X-ray crystallography or NMR spectroscopy. 3D structure obtained from Protein Data Bank. Autodock program helps to predict interaction between target and ligand with refer to geometric position of reactive groups, bond angles,bond length, side chain flexibility and molecular force fields.