Applications of genetic engineering and biotechnology I • Genetic engineering, Genetic modification
(KCSPK 22) - The ability to manipulate DNA in vitro and to introduce
genes into living cells has allowed scientists to generate
Biotechnology applications
new varieties of plants, animals, and other organisms
This chapter will examine the applications of Genetic Engineering with specific traits
and Biotechnology in medicine and agriculture, and conclude by - Genetic engineering typically involves the use of
considering some ethical issues that arose from the use of the recombinant DNA technologies to add a gene or genes
technology. to a genome, but it can also involve gene removal
- These organisms are called genetically modified
The chapter will be presented in six lectures:
organisms (GMOs)
1. Biopharmaceutical products and vaccines • Genome editing
2. GM plants and animals I - Genome editing can potentially edit DNA in vivo to
3. GM plants and animals II introduce specific mutations in a very precise manner,
4. Genetic testing and medical diagnostics without introducing additional DNA into the recipient
5. Genetic analysis of individual genomes and single cells; organism
Genome-wide association studies (GWAS)
6. Synthetic Biology (SynBio) and Ethics (and the future) Biopharmaceutical products and vaccines
Genetic engineering, biotechnology (and genome editing) • GMOs are used extensively in the pharmaceutical industry
What is in a name? GM vs. GE for the production of a variety of products, especially
therapeutic proteins to treat diseases
• Biotechnology • Biopharming produces valuable proteins in genetically
- Uses living organisms to create products or processes modified (GM) animals and plants
that help improve the quality of life for humans or other
organisms
- Is a science dating back to ancient civilizations when
microbes were used to make products like wine and
beer, vinegar, breads, and cheeses
- Modern Biotech began shortly after recombinant DNA
technology was developed.
- Has since exploded to become a modern multi-billion
dollar industry the has revolutionized medicine,
agriculture and several other industries
,Recombinant protein production in bacteria Biopharmaceutical production in transgenic animal hosts
• The first commercial biotech product, manufactured by • Bacterial expression systems often fail to properly process
recombinant DNA technology, was human insulin, called the post-translational modifications (like glycosylation or
Humulin®, was licensed for therapeutic use in 1982 phosphorylation) required for eukaryotic proteins to be fully
• Previously, insulin was chemically extracted from the functional
pancreas of cows and pigs at abattoirs • In addition, eukaryotic proteins produced in bacterial cells
• Shortly after insulin, the human growth hormone gene was often do not fold into the proper three-dimensional
cloned, and turned into a commercial product to treat conformation required for functionality
children who su[er from a form of dwarfism • Transgenic animal systems can solve these problems –
• Many therapeutic proteins have since been produced by examples are yeast cells, insect cell lines, or transgenic
expressing human genes in bacteria animals, which can act as living ‘bioreactors’ or
• In most cases, the human gene is cloned into a plasmid, ‘biofactories’.
and the recombinant vector is introduced into the bacterial - In 2006, antithrombin (anticlotting protein in blood)
host, which are grown in large industrial fermenters, and became the first drug to be produced in the milk of farm
the recombinant human proteins recovered and purified animals, after the human antithrombin gene was
from these bacterial cultures expressed in the mammary glands of goats
- In one year, a single goat will produce the equivalent
amount of antithrombin that would require ∼90,000
human blood collections
- Chickens are easier to rear and more e[icient as
biofactories– very e[icient in glycosylating recombinant
proteins.
- Kanuma is made from the eggs of GM chickens
expressing a recombinant form of the enzyme
lysosomal acid lipase (LAL), or sebelipase alfa, that is
used as a medication for the treatment of lysosomal
acid lipase deficiency
, • In 2014, the National Department of Health introduced HPV
vaccination as a central strategy for cervical cancer
prevention in South Africa. Cervical cancer is the 2nd most
common cancer among South African women
Vaccine production in plants
• Plants o[er several advantages for expressing recombinant
proteins
- Once a transgenic plant is created, it can easily be
Recombinant vaccine production grown and vegetatively propagated in a greenhouse or
• Vaccines field to provide a constant source of recombinant
- Inactivated vaccines are prepared from ‘killed’ versions protein
of the infectious virus or bacteria, e.g. Rabies and - The cost of expressing a recombinant protein in a
Influenza transgenic plant is much lower than making the same
- Attenuated vaccines, which are ‘live’ viruses or bacteria protein in bacteria, yeast, or mammalian cells
that can no longer reproduce but can cause a mild form - Antibodies against Ebolavirus were expressed in
of the disease, e.g. Tuberculosis, cholera, and tobacco leaves. Ebola monoclonal antibody genes from
chickenpox transgenic mice were introduced into tobacco plants,
• Genetic engineering is being used to produce subunit which expressed high quantities of the antibody
vaccines, which consist of one or more antigenic surface proteins. These can be isolated and purified for use in
proteins from the virus or bacterium rather than the entire humans
- Disadvantages are temperature sensitivity and sterile
virus or bacterium, e.g. Johnson&Johnson SARS- Cov-2
vaccine. conditions for administering
- In 2005, the FDA approved Gardasil®, a subunit vaccine - Edible vaccines – expressing a vaccine in a fruit or
vegetable
that targets four strains of human papillomavirus (HPV)
that cause cervical cancers o Cholera in potatoes
o Hepatitis B in bananas
(KCSPK 22) - The ability to manipulate DNA in vitro and to introduce
genes into living cells has allowed scientists to generate
Biotechnology applications
new varieties of plants, animals, and other organisms
This chapter will examine the applications of Genetic Engineering with specific traits
and Biotechnology in medicine and agriculture, and conclude by - Genetic engineering typically involves the use of
considering some ethical issues that arose from the use of the recombinant DNA technologies to add a gene or genes
technology. to a genome, but it can also involve gene removal
- These organisms are called genetically modified
The chapter will be presented in six lectures:
organisms (GMOs)
1. Biopharmaceutical products and vaccines • Genome editing
2. GM plants and animals I - Genome editing can potentially edit DNA in vivo to
3. GM plants and animals II introduce specific mutations in a very precise manner,
4. Genetic testing and medical diagnostics without introducing additional DNA into the recipient
5. Genetic analysis of individual genomes and single cells; organism
Genome-wide association studies (GWAS)
6. Synthetic Biology (SynBio) and Ethics (and the future) Biopharmaceutical products and vaccines
Genetic engineering, biotechnology (and genome editing) • GMOs are used extensively in the pharmaceutical industry
What is in a name? GM vs. GE for the production of a variety of products, especially
therapeutic proteins to treat diseases
• Biotechnology • Biopharming produces valuable proteins in genetically
- Uses living organisms to create products or processes modified (GM) animals and plants
that help improve the quality of life for humans or other
organisms
- Is a science dating back to ancient civilizations when
microbes were used to make products like wine and
beer, vinegar, breads, and cheeses
- Modern Biotech began shortly after recombinant DNA
technology was developed.
- Has since exploded to become a modern multi-billion
dollar industry the has revolutionized medicine,
agriculture and several other industries
,Recombinant protein production in bacteria Biopharmaceutical production in transgenic animal hosts
• The first commercial biotech product, manufactured by • Bacterial expression systems often fail to properly process
recombinant DNA technology, was human insulin, called the post-translational modifications (like glycosylation or
Humulin®, was licensed for therapeutic use in 1982 phosphorylation) required for eukaryotic proteins to be fully
• Previously, insulin was chemically extracted from the functional
pancreas of cows and pigs at abattoirs • In addition, eukaryotic proteins produced in bacterial cells
• Shortly after insulin, the human growth hormone gene was often do not fold into the proper three-dimensional
cloned, and turned into a commercial product to treat conformation required for functionality
children who su[er from a form of dwarfism • Transgenic animal systems can solve these problems –
• Many therapeutic proteins have since been produced by examples are yeast cells, insect cell lines, or transgenic
expressing human genes in bacteria animals, which can act as living ‘bioreactors’ or
• In most cases, the human gene is cloned into a plasmid, ‘biofactories’.
and the recombinant vector is introduced into the bacterial - In 2006, antithrombin (anticlotting protein in blood)
host, which are grown in large industrial fermenters, and became the first drug to be produced in the milk of farm
the recombinant human proteins recovered and purified animals, after the human antithrombin gene was
from these bacterial cultures expressed in the mammary glands of goats
- In one year, a single goat will produce the equivalent
amount of antithrombin that would require ∼90,000
human blood collections
- Chickens are easier to rear and more e[icient as
biofactories– very e[icient in glycosylating recombinant
proteins.
- Kanuma is made from the eggs of GM chickens
expressing a recombinant form of the enzyme
lysosomal acid lipase (LAL), or sebelipase alfa, that is
used as a medication for the treatment of lysosomal
acid lipase deficiency
, • In 2014, the National Department of Health introduced HPV
vaccination as a central strategy for cervical cancer
prevention in South Africa. Cervical cancer is the 2nd most
common cancer among South African women
Vaccine production in plants
• Plants o[er several advantages for expressing recombinant
proteins
- Once a transgenic plant is created, it can easily be
Recombinant vaccine production grown and vegetatively propagated in a greenhouse or
• Vaccines field to provide a constant source of recombinant
- Inactivated vaccines are prepared from ‘killed’ versions protein
of the infectious virus or bacteria, e.g. Rabies and - The cost of expressing a recombinant protein in a
Influenza transgenic plant is much lower than making the same
- Attenuated vaccines, which are ‘live’ viruses or bacteria protein in bacteria, yeast, or mammalian cells
that can no longer reproduce but can cause a mild form - Antibodies against Ebolavirus were expressed in
of the disease, e.g. Tuberculosis, cholera, and tobacco leaves. Ebola monoclonal antibody genes from
chickenpox transgenic mice were introduced into tobacco plants,
• Genetic engineering is being used to produce subunit which expressed high quantities of the antibody
vaccines, which consist of one or more antigenic surface proteins. These can be isolated and purified for use in
proteins from the virus or bacterium rather than the entire humans
- Disadvantages are temperature sensitivity and sterile
virus or bacterium, e.g. Johnson&Johnson SARS- Cov-2
vaccine. conditions for administering
- In 2005, the FDA approved Gardasil®, a subunit vaccine - Edible vaccines – expressing a vaccine in a fruit or
vegetable
that targets four strains of human papillomavirus (HPV)
that cause cervical cancers o Cholera in potatoes
o Hepatitis B in bananas
