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Role of Biotechnology in The
Management of Disease
Gopal Jee Gopal, PhD(JNU)
Assistant Professor
C.G.Bhakta Institute of Biotechnology
Uka Tarsadia University, Bardoli
What is Biotechnology?
Using living organism or materials for commercial, industrial & healthcare
purpose(human welfare).
Biotechnology is the use of Biological processes, organisms or systems to
manufacture products intended to improve the quality of human life.
Gene manipulation technology/Recombinant DNA Technology/Gene cloning
has revolutionized biotechnology.
Gene: A gene is the basic functional unit of heredity.
Genes are segments of DNA and having information in the form of
nucleotide sequence which is decoded, when required, in the form of
mRNA /proteins.
Gene , directly or indirectly, is responsible for every character of an
organism.
5’-ATGCCTAGGTACCTATGA-3’
3’-TACGGATCCATGGATACT-5’
5’-AUG CCU AGG UAC CUA UGA-3’
N-MET-PRO-ARG-TYR-LEU-C
DNA/gene
Transcription
Translation
mRNA
Protein
Flow of information coded in the gene
Basis of Gene Manipulation technology
* Codons and decoding pathways are almost universal.
Overview of recombinant DNA technology/Gene manipulation
Bacterial cell
Bacterial
chromosome Plasmid
Gene of interest
DNA containing
gene of interest
Isolate plasmid.
Enzymatically cleave DNA into fragments.
Isolate fragment
with the gene of
interest.
Ligate gene into plasmid.
Transform bacteria with ligated plasmid
Culture bacteria.
Harvest copies of gene to insert into
plants or animals
Harvest proteins coded by gene
Application of Gene Manipulation
Technology in the Management of
Diseases.
1. Nutritious food to overcome disease caused by nutrition
deficiency.
2. Prevention of Pathogenic Disease by Vaccination/
immunization
3. Diagnosis of Disease:
4. Treatment of Disease:
5. Understanding the new/unknown disease.
1. How Biotechnology(Gene
Manipulation) helps in Providing
Nutritious food?
1. Golden Rice: a transgenic rice having high Vitamin A content.
Carotene( precursor of VitA) biosynthetic gene has been introduced
in paddy.
2. High iron Containing transgenic Banana:
Ferritin gene has been introduced.
3. Transgenic soya with high amount of Vit E.
2. How Biotechnology helps in
Prevention of Disease?
Production of Different categories of vaccines: Vaccination is the
administration of antigenic material (Vaccine) to stimulate an
individual's immune system to develop immunity against pathogen.
Types of vaccine: Attenuated Vaccine , inactivated vaccine,
Subunit vaccine, DNA Vaccine etc.
Use of virus coat protein as vaccine
T.A. Brawn
Vaccination
Principle of Gene Manipulation 4th ed..
3. Biotechnology in Diagnosis of
Disease.
1. PCR based Diagnosis of disease: PCR, RT- PCR and Real time
PCR are used to diagnose diseases. Presence of bacteria/
virus can be detected at early stage of infection.
2. Antibody based Diagnosis of Disease: eg. ELISA,
Immunocytochemistry, immunohistochemistry, Western blotting based,
Radioimmuno assay, Immunoflourescence, immunoelectron microscopy etc.
3. DNA hybridization based diagnosis: Southern blotting, Northern
Blotting, FISH , DNA micro array etc.
4. DNA sequence based Diagnosis: Single Nucleotide
polymorphism. Gene sequencing, Finding triplet codon repeats
( huntington’s disease).
PCR based Diagnosis
Invitro amplification of a known ( at least terminal seq should be
known) DNA segments.
A. Increases quantity to million-billion times .
B. Amplify only specific sequence from mixture of DNA.
R.T PCR and Real time PCR are used to observe expression of gene/s so can
be used to observe presence of pathogenic RNA viruses too.
Virus load can also be quantified.
Immunofluorescence tech
WWW. Cellsignal.com
4. Treatment of Diseases
A. Therapeutic Recombinant Proteins: Several human disorders are due to
absence of or malfunctioning of protein usually synthesized in the body.
Human proteins are produced in bacteria or other organism using recombinant
DNA technology
eg. Insulin (humulin), Growth hormone, blood clotting factor etc.
B. Therapeutic nucleic acid used for Gene Therapy:
Addition or deletion of gene which cause disease or mask the expression of
disease causing gene by antisense technology.
C. Therapeutic antibodies: Antibodies conjugated with toxin kills cancer
cell/tissue.
D. Antibiotics: Use to treat microbial disease. Antibiotics are secreted by
Microorganism but to increase yield or get modified version of antibiotics
scientists apply gene manipulation technology.
E. Biosynthesis and production of natural drugs are being increase applying
metabolic engineering. Eg. Vinblastine, vincristine etc.
A. Recombinant Therapeutic protein
T.A. Brawn
B. Gene Therapy
This is a technique whereby a working gene replaces the absent or faulty gene,
so that the body can make the correct enzyme or protein and consequently
eliminate the root cause of the disease. Gene therapy may also used for curing
Cancer.
Ex-Vivo Gene Therapy
Principle of Gene manipulation 4th ed.
C. Therapeutic Monoclonal antibody
Name of Monoclonal
antibody
Target Target disease
abciximab GPIIb/IIIa Percutaneous coronary intervention
adalimumab TNF Rheumatoid arthritis
adalimumab-atto TNF Crohn's disease
Ulcerative colitis
Plaque psoriasis
ado-trastuzumab
emtansine
HER2 Metastatic breast cancer
alemtuzumab CD52 B-cell chronic lymphocytic leukemia
alirocumab PCSK9 Heterozygous familial
hypercholesterolemia
Refractory
hypercholesterolemia
atezolizumab PD-L1 Urothelial carcinoma
basiliximab IL2RA Prophylaxis of acute organ
rejectionin renal transplant
daclizumab IL2R Multiple sclerosis
Production of Monoclonal Antibody
D.Impact of discovery of Antibiotics
It is estimated that penicillin has saved at least
200 million lives since its first use as a medicine in
1942.
E.Increasing Drug biosynthesis and
production by metabolic engineering.
• Removal of rate-limiting transcriptional and allosteric
controls.
• Kinetic enhancement of rate-limiting enzymes.
• Genetic inhibition of competing pathways.
• Enhanced carbon commitment to the primary metabolic
pathway from central metabolism.
• Modification of secondary metabolic pathways to
enhance energy metabolism and the availability
of enzymatic cofactors.
• Enhanced transport of the compound out of the
cell.
5. To understand the cause and
Mechanism of Disease
1. Transgenic animals as models of Human disease:
We create diseased mice, rabbit by deletion or addition of gene to understand
the disease.
Ex: A.Creation of model of Alzheimer’s disease by overexpression of
Amyloid precursor Protein.
B. Creation of mouse model ( by knock out) for hypoxanthine-guanine
phosphoribosyltransferase (HPRT) deficiency.
C. Gene targeting has been widely used to model human cancers caused by the
inactivation ( Gene knock out) of Tumour suppressor genes such as TP53 and
RB1.
2. Finding the function/role of unknown gene: Knock out, knock down,
overexpression.
3. Pharmacogenomics: Role of genome in drug response.
It opens the field personalised medicine.
Summary
Gene manipulation technology in
Disease Management
1.Nutritious
food
Eg. Golden rice,
Iron containing
Banana.
2.Prevention
Immunization
eg. Vaccine against
pathogen
3. Diagnosis
Eg. PCR based ,
Blotting based
ELISA based
Western blotting
based.
4.Cure/treatment
Therapeutic proteins,
oligonucleotide
Antibodies and
Antibiotics.
5. Understanding
the disease:
Creation of diseased
Animal model.
Pharmacogenomics
Thank You
For
Attention
Send your feedback at
gopaljigopal@rediffmail.com
Gopal.jee@utu.ac.in
or at 9558880617, 8160245501

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Role of biotechnology in the management of disease

  • 1. Role of Biotechnology in The Management of Disease Gopal Jee Gopal, PhD(JNU) Assistant Professor C.G.Bhakta Institute of Biotechnology Uka Tarsadia University, Bardoli
  • 2. What is Biotechnology? Using living organism or materials for commercial, industrial & healthcare purpose(human welfare). Biotechnology is the use of Biological processes, organisms or systems to manufacture products intended to improve the quality of human life. Gene manipulation technology/Recombinant DNA Technology/Gene cloning has revolutionized biotechnology. Gene: A gene is the basic functional unit of heredity. Genes are segments of DNA and having information in the form of nucleotide sequence which is decoded, when required, in the form of mRNA /proteins. Gene , directly or indirectly, is responsible for every character of an organism.
  • 3. 5’-ATGCCTAGGTACCTATGA-3’ 3’-TACGGATCCATGGATACT-5’ 5’-AUG CCU AGG UAC CUA UGA-3’ N-MET-PRO-ARG-TYR-LEU-C DNA/gene Transcription Translation mRNA Protein Flow of information coded in the gene Basis of Gene Manipulation technology * Codons and decoding pathways are almost universal.
  • 4. Overview of recombinant DNA technology/Gene manipulation Bacterial cell Bacterial chromosome Plasmid Gene of interest DNA containing gene of interest Isolate plasmid. Enzymatically cleave DNA into fragments. Isolate fragment with the gene of interest. Ligate gene into plasmid. Transform bacteria with ligated plasmid Culture bacteria. Harvest copies of gene to insert into plants or animals Harvest proteins coded by gene
  • 5. Application of Gene Manipulation Technology in the Management of Diseases. 1. Nutritious food to overcome disease caused by nutrition deficiency. 2. Prevention of Pathogenic Disease by Vaccination/ immunization 3. Diagnosis of Disease: 4. Treatment of Disease: 5. Understanding the new/unknown disease.
  • 6. 1. How Biotechnology(Gene Manipulation) helps in Providing Nutritious food? 1. Golden Rice: a transgenic rice having high Vitamin A content. Carotene( precursor of VitA) biosynthetic gene has been introduced in paddy. 2. High iron Containing transgenic Banana: Ferritin gene has been introduced. 3. Transgenic soya with high amount of Vit E.
  • 7. 2. How Biotechnology helps in Prevention of Disease? Production of Different categories of vaccines: Vaccination is the administration of antigenic material (Vaccine) to stimulate an individual's immune system to develop immunity against pathogen. Types of vaccine: Attenuated Vaccine , inactivated vaccine, Subunit vaccine, DNA Vaccine etc.
  • 8. Use of virus coat protein as vaccine T.A. Brawn
  • 9. Vaccination Principle of Gene Manipulation 4th ed..
  • 10.
  • 11. 3. Biotechnology in Diagnosis of Disease. 1. PCR based Diagnosis of disease: PCR, RT- PCR and Real time PCR are used to diagnose diseases. Presence of bacteria/ virus can be detected at early stage of infection. 2. Antibody based Diagnosis of Disease: eg. ELISA, Immunocytochemistry, immunohistochemistry, Western blotting based, Radioimmuno assay, Immunoflourescence, immunoelectron microscopy etc. 3. DNA hybridization based diagnosis: Southern blotting, Northern Blotting, FISH , DNA micro array etc. 4. DNA sequence based Diagnosis: Single Nucleotide polymorphism. Gene sequencing, Finding triplet codon repeats ( huntington’s disease).
  • 12. PCR based Diagnosis Invitro amplification of a known ( at least terminal seq should be known) DNA segments. A. Increases quantity to million-billion times . B. Amplify only specific sequence from mixture of DNA. R.T PCR and Real time PCR are used to observe expression of gene/s so can be used to observe presence of pathogenic RNA viruses too. Virus load can also be quantified.
  • 14. 4. Treatment of Diseases A. Therapeutic Recombinant Proteins: Several human disorders are due to absence of or malfunctioning of protein usually synthesized in the body. Human proteins are produced in bacteria or other organism using recombinant DNA technology eg. Insulin (humulin), Growth hormone, blood clotting factor etc. B. Therapeutic nucleic acid used for Gene Therapy: Addition or deletion of gene which cause disease or mask the expression of disease causing gene by antisense technology. C. Therapeutic antibodies: Antibodies conjugated with toxin kills cancer cell/tissue. D. Antibiotics: Use to treat microbial disease. Antibiotics are secreted by Microorganism but to increase yield or get modified version of antibiotics scientists apply gene manipulation technology. E. Biosynthesis and production of natural drugs are being increase applying metabolic engineering. Eg. Vinblastine, vincristine etc.
  • 15. A. Recombinant Therapeutic protein T.A. Brawn
  • 16. B. Gene Therapy This is a technique whereby a working gene replaces the absent or faulty gene, so that the body can make the correct enzyme or protein and consequently eliminate the root cause of the disease. Gene therapy may also used for curing Cancer.
  • 17.
  • 18. Ex-Vivo Gene Therapy Principle of Gene manipulation 4th ed.
  • 19. C. Therapeutic Monoclonal antibody Name of Monoclonal antibody Target Target disease abciximab GPIIb/IIIa Percutaneous coronary intervention adalimumab TNF Rheumatoid arthritis adalimumab-atto TNF Crohn's disease Ulcerative colitis Plaque psoriasis ado-trastuzumab emtansine HER2 Metastatic breast cancer alemtuzumab CD52 B-cell chronic lymphocytic leukemia alirocumab PCSK9 Heterozygous familial hypercholesterolemia Refractory hypercholesterolemia atezolizumab PD-L1 Urothelial carcinoma basiliximab IL2RA Prophylaxis of acute organ rejectionin renal transplant daclizumab IL2R Multiple sclerosis
  • 21. D.Impact of discovery of Antibiotics It is estimated that penicillin has saved at least 200 million lives since its first use as a medicine in 1942.
  • 22. E.Increasing Drug biosynthesis and production by metabolic engineering. • Removal of rate-limiting transcriptional and allosteric controls. • Kinetic enhancement of rate-limiting enzymes. • Genetic inhibition of competing pathways. • Enhanced carbon commitment to the primary metabolic pathway from central metabolism. • Modification of secondary metabolic pathways to enhance energy metabolism and the availability of enzymatic cofactors. • Enhanced transport of the compound out of the cell.
  • 23. 5. To understand the cause and Mechanism of Disease 1. Transgenic animals as models of Human disease: We create diseased mice, rabbit by deletion or addition of gene to understand the disease. Ex: A.Creation of model of Alzheimer’s disease by overexpression of Amyloid precursor Protein. B. Creation of mouse model ( by knock out) for hypoxanthine-guanine phosphoribosyltransferase (HPRT) deficiency. C. Gene targeting has been widely used to model human cancers caused by the inactivation ( Gene knock out) of Tumour suppressor genes such as TP53 and RB1. 2. Finding the function/role of unknown gene: Knock out, knock down, overexpression. 3. Pharmacogenomics: Role of genome in drug response. It opens the field personalised medicine.
  • 24. Summary Gene manipulation technology in Disease Management 1.Nutritious food Eg. Golden rice, Iron containing Banana. 2.Prevention Immunization eg. Vaccine against pathogen 3. Diagnosis Eg. PCR based , Blotting based ELISA based Western blotting based. 4.Cure/treatment Therapeutic proteins, oligonucleotide Antibodies and Antibiotics. 5. Understanding the disease: Creation of diseased Animal model. Pharmacogenomics
  • 25. Thank You For Attention Send your feedback at gopaljigopal@rediffmail.com Gopal.jee@utu.ac.in or at 9558880617, 8160245501