SlideShare a Scribd company logo
1 of 31
Download to read offline
GRACIOUS COLLEGE OF NURSING
ABHANPUR RAIPUR C.G
HUMAN GENOME PROJECT
AND
GENE THERAPY
GENE THERAPY
PRESENTED BY
OM VERMA
ASSISTANT PROFESSOR
GENOME
Genome is the complete DNA sequence
contained in the chromosomes of an
organism which carries its complete
genetic information. The human
genome is a complete set of nucleic acid
genome is a complete set of nucleic acid
sequences for humans,
In Humans, the genome consists of 23
pairs of chromosomes located in the
cell's nucleus,
Human Genome Project :- It is a mega project
was an international scientific research project
with the goal of determining the base pairs that
make up human DNA, and of identifying,
mapping and sequencing all of the genes of
HUMAN GENOME PROJECT
mapping and sequencing all of the genes of
the human genome from both a physical and a
functional viewpoint. It started in 1990 and was
completed in 2003. 13 Year project. under
Dr. james D Watson at the United States
National Institute of Health .
DEFINITION
Human Genome Project was an
international research effort to
determine the sequence of the human
genome and identify the genes that it
genome and identify the genes that it
contains.
According to S.S Randhawa
GOALS –
1. Identify all the 20,000 – 25,000 ( approx ) gens in Human
DNA. ( counting of alleles low and multiples of alleles
and their gens locus )
2. Determine the sequences of the 3 billion chemical base
2. Determine the sequences of the 3 billion chemical base
pairs that make up human DNA.
3. Develop software for large scale DNA analysis ,store all
found information in data base and improve tools for
data analysis.
4. Collect and distribute .
5. Transfer related technologies to the private sectors .
6. Improve the sequencing technology by developing
New and more effective methods
7. Analyze sequence variations in the human genome,
such as single nucleotide polymorphisms ( the
such as single nucleotide polymorphisms ( the
presence of two or more variant forms of a specific
DNA sequence ) and other DNA sequence variations’
8. To map all of human gene
9. To find total number of gene
1o. Study the ethical ,legal and social Issues
genetic ( right and wrong ) research that
may arise from the project.
USES OF HUMAN GENOME –
1. Etiology of cancers, Alzheimer's
disease etc.
2. Defining the pathogenesis of a
2. Defining the pathogenesis of a
disease and to study the disease
process at molecular
3. Sensitivity, of an individual to a
variety of illness. level.
4. Precise and accurate, new ways to prevent a
Number of diseases that affect human beings.
5. To diagnose and treat disease. Target genes
for treatment and management of disease.
6. Human development and anthropology.
( human societies )
7. Improving knowledge on mutation .
GENE THERAPY
GENE THERAPY
INTRODUCTION
Gene therapy is a medical field which focuses
on the genetic modification of cells to produce
a therapeutic effect or the treatment of
disease by repairing or reconstructing defective
disease by repairing or reconstructing defective
genetic material. healthy gene may replace a
damaged (mutated) gene, inactivate a mutated
gene or introduce an entirely new gene.
Gene therapy is a define as the
replacement of a deficiency gene
product or repair of abnormal gene
within an individual’s cells and
tissue to treat disease.
tissue to treat disease.
According to Siddhartha Mukherjee
Gene therapy is a technique
which involves the replacement
of defective genes with healthy
ones in order to treat genetic
ones in order to treat genetic
disorders.
According to AND
Gene therapy is the insertion of genes
into an individuals cells and tissue to
treat disease and hereditary disease
and treatment in which the genes that
are missing or not normal in cell are
are missing or not normal in cell are
replace with normal genes that
condition is called gene therapy
According to V.K. Agarwal
Applications of gene therapy:
Gene therapy should only be
used to treat serious diseases
used to treat serious diseases
The various applications are:
INTRODUCTION OF NORMALLY
FUNCTIONING GENE:
The most common form of gene therapy
involves the insertion of "normal" functional
genes into the genome. When there is
genes into the genome. When there is
defective gene in the cells, it can not produce
the respective protein which normal gene
produces. In order to produce a protein
product, the defective gene causing the disease
is replaced by a healthy copy of the gene.
INTRODUCTION OF TOXIC GENE:
Genes whose products are toxic may be introduced to
cause direct death of malignant cells or infective
organisms in the cells.
INTRODUCTION OF PRODRUG GENE: The products of
introduced genes may act as prodrugs and kill the cells
by conventionally administered cytotoxic agents.
by conventionally administered cytotoxic agents.
INTRODUCTION OF ANTIGEN OR CYTOKINE GENE:
This may stimulate immune response against neoplastic
cells or infective disease and result in immune mediated
destruction.
INACTIVITY/KNOCKING OUT GENE:
The non functional defective gene may
be removed by this approach.
TYPES
OF
OF
GENE THERAPY
The cells in the body can be
divided into two main categories :-
1. Somatic Cell Therapy
1. Somatic Cell Therapy
2. Germ Cell Therapy
1. SOMATIC CELL THERAPY Somatic cell gene therapy
involves the placement of a human gene into a living
person's somatic cells—cells that do not produce the
eggs and sperm that in turn produce the next generation.
Transfer of genes into the somatic cells of the patients as
cell of bone marrow Somatic cell gene therapy would
cell of bone marrow Somatic cell gene therapy would
aim to cure a disease only in the patient, Somatic gene
transfer is carried out by two methods, such as
1. IN VIVO ( a process) performed or taking place in a living organism.).
2. EX VIVO (outside the body or in the laboratory).
1. IN VIVO
IN VIVO :- In vivo gene therapy, the therapeutic genetic
material is introduced directly into the patients body
new genes are inserted directly into body. is the
insertion of functioning genes directly to the human
body through the bloodstream.
2. EX VIVO
Ex vivo :- Gene therapy involves removing cells from
body, genetically modification ex vivo in a
laboratory. , and placing and transfer them back into
patient body.
2. GERM CELL THERAPY-
Germline gene therapy is when DNA is transferred
into the cells that produce reproductive cells, eggs
or sperm, in the body. This type of therapy allows
for the correction of disease-causing gene variants
for the correction of disease-causing gene variants
that are certain to be passed down from generation
to generation. Germ cell therapy in germ cell are
used and it result permanent change that are
passed on to the future generations .
METHOD OF GENE THERAPY
METHOD OF GENE THERAPY
VECTORS IN GENE THERAPY
The most common form of gene therapy
involves of normal gene into the genome
with the help of certain carriers called
vectors
vectors
There are two vectors are used in gene
therapy
1. VIRAL VECTORS
2. NON VIRAL VECTORS
1.VIRAL VECTORS :-
They are the most commonly used vectors.
These viruses are genetically modified
such that they do not to cause disease, in
patients who receive gene therapy. Various
patients who receive gene therapy. Various
viruses that can be used as vectors for
gene therapy include: retroviruses,
adenoviruses, herpes simplex viruses.
2. NON VIRAL VECTORS
Non viral method: Gene delivery can also
be carried by non viral methods. The non-
viral vectors are Naked DNA, particle
based and chemical based.
based and chemical based.
Method:- Direct introduction The
therapeutic naked DNA is directly
introduced into target cells.
Liposome mediated DNA transfer :
liposome mediated gene delivery is the
way of transferring gene into the liposome
to mediate gene into the cell and perform
its desired function. liposome mediated
gene delivery is the way of transferring
gene delivery is the way of transferring
gene into the liposome to mediate gene
into the cell and perform its desired
function.
Human artificial chromosome
Stem cell of bone marrow
ADVANTAGE OF GENE THERAPY
1. Eliminate and prevent hereditary disease.
2. It help to eliminate disease from future
generation.
3. By this disease therapy possible to cure the
heart disease or aids and cancer .
HUMAN GENOME PROJECT AND GENE THERAPY.pdf

More Related Content

What's hot

What's hot (20)

Gen therapy
Gen therapyGen therapy
Gen therapy
 
Human Genome Project
Human Genome ProjectHuman Genome Project
Human Genome Project
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Human genome project
Human genome projectHuman genome project
Human genome project
 
Human genome project
Human genome projectHuman genome project
Human genome project
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Human Genome Project
Human Genome ProjectHuman Genome Project
Human Genome Project
 
Cancer genetics
Cancer geneticsCancer genetics
Cancer genetics
 
molecular medicine.pptx
molecular medicine.pptxmolecular medicine.pptx
molecular medicine.pptx
 
Human genetic technologies
Human genetic technologiesHuman genetic technologies
Human genetic technologies
 
Genetic testing
Genetic testingGenetic testing
Genetic testing
 
Human genome project
Human genome projectHuman genome project
Human genome project
 
Medical Biotechnology (Recent Development)
Medical Biotechnology (Recent Development)Medical Biotechnology (Recent Development)
Medical Biotechnology (Recent Development)
 
Sanger sequencing method of DNA
Sanger sequencing method of DNA Sanger sequencing method of DNA
Sanger sequencing method of DNA
 
Gene transfer technologies
Gene transfer technologiesGene transfer technologies
Gene transfer technologies
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Gene theapy
Gene theapyGene theapy
Gene theapy
 
Chapter 1 cancer genome
Chapter 1 cancer genomeChapter 1 cancer genome
Chapter 1 cancer genome
 
Gene therapy
Gene therapy Gene therapy
Gene therapy
 
Cancer epigenetics
Cancer epigeneticsCancer epigenetics
Cancer epigenetics
 

Similar to HUMAN GENOME PROJECT AND GENE THERAPY.pdf

Similar to HUMAN GENOME PROJECT AND GENE THERAPY.pdf (20)

Gene therapy
Gene therapyGene therapy
Gene therapy
 
gene therapy.
gene therapy.gene therapy.
gene therapy.
 
Gene Therapy
Gene Therapy Gene Therapy
Gene Therapy
 
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptx
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptxNUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptx
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptx
 
Gene therapy.pptxjjjjnnnsnskskslsllssnnznzj
Gene therapy.pptxjjjjnnnsnskskslsllssnnznzjGene therapy.pptxjjjjnnnsnskskslsllssnnznzj
Gene therapy.pptxjjjjnnnsnskskslsllssnnznzj
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
gene therapy notes: introduction, working , types
gene therapy notes: introduction, working , typesgene therapy notes: introduction, working , types
gene therapy notes: introduction, working , types
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Genetic Engineering
Genetic Engineering Genetic Engineering
Genetic Engineering
 
GENE THERAPY : introduction, advancements and applications
GENE THERAPY : introduction, advancements and applicationsGENE THERAPY : introduction, advancements and applications
GENE THERAPY : introduction, advancements and applications
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
GENE THERAPY.docx
GENE THERAPY.docxGENE THERAPY.docx
GENE THERAPY.docx
 
GENE THERAPY
GENE THERAPYGENE THERAPY
GENE THERAPY
 
Nucleic Acid Based Therapeutic Delivery System.pptx
Nucleic Acid Based Therapeutic Delivery System.pptxNucleic Acid Based Therapeutic Delivery System.pptx
Nucleic Acid Based Therapeutic Delivery System.pptx
 
Gene therapy presentation
Gene therapy presentationGene therapy presentation
Gene therapy presentation
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Gene Therapy by Anupam Dwivedi (MS)
Gene Therapy by  Anupam Dwivedi (MS)Gene Therapy by  Anupam Dwivedi (MS)
Gene Therapy by Anupam Dwivedi (MS)
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
GENE THERAPHY
GENE THERAPHYGENE THERAPHY
GENE THERAPHY
 

More from OM VERMA

More from OM VERMA (20)

otalgia- JUN 2023 OM VERMA.pdf
otalgia- JUN 2023 OM VERMA.pdfotalgia- JUN 2023 OM VERMA.pdf
otalgia- JUN 2023 OM VERMA.pdf
 
POISIONING JUN 2023
POISIONING JUN 2023POISIONING JUN 2023
POISIONING JUN 2023
 
public relation.pdf
public relation.pdfpublic relation.pdf
public relation.pdf
 
PRENATAL TESTING AND DIAGNOSIS.pdf
PRENATAL TESTING AND DIAGNOSIS.pdfPRENATAL TESTING AND DIAGNOSIS.pdf
PRENATAL TESTING AND DIAGNOSIS.pdf
 
practical application of genetics in nursing OM VERMA 2022 slide.pdf
practical application of genetics in nursing  OM VERMA 2022 slide.pdfpractical application of genetics in nursing  OM VERMA 2022 slide.pdf
practical application of genetics in nursing OM VERMA 2022 slide.pdf
 
Neural tube defects and the role of folic acid in Lowering the Risk.pdf
Neural tube defects and the role of folic acid in Lowering the Risk.pdfNeural tube defects and the role of folic acid in Lowering the Risk.pdf
Neural tube defects and the role of folic acid in Lowering the Risk.pdf
 
MULTIPLE ALLOTS ( ALLELES ) AND BLOOD GROUPS OM VERMA 2023.pdf
MULTIPLE ALLOTS  ( ALLELES ) AND BLOOD GROUPS OM VERMA 2023.pdfMULTIPLE ALLOTS  ( ALLELES ) AND BLOOD GROUPS OM VERMA 2023.pdf
MULTIPLE ALLOTS ( ALLELES ) AND BLOOD GROUPS OM VERMA 2023.pdf
 
MENDALIAN THEORY OF INHERITANCE AND MULTIPLE BLOOD ALLOTS AND BLOOD GROUPS 20...
MENDALIAN THEORY OF INHERITANCE AND MULTIPLE BLOOD ALLOTS AND BLOOD GROUPS 20...MENDALIAN THEORY OF INHERITANCE AND MULTIPLE BLOOD ALLOTS AND BLOOD GROUPS 20...
MENDALIAN THEORY OF INHERITANCE AND MULTIPLE BLOOD ALLOTS AND BLOOD GROUPS 20...
 
Maternal prenatal and genetic influences on development of defect and disease...
Maternal prenatal and genetic influences on development of defect and disease...Maternal prenatal and genetic influences on development of defect and disease...
Maternal prenatal and genetic influences on development of defect and disease...
 
MATERNAL AGE,MATERNAL DRUG THERAPY PRENATAL TEST AND DIAGNOSIS.pdf
MATERNAL AGE,MATERNAL DRUG THERAPY PRENATAL TEST AND DIAGNOSIS.pdfMATERNAL AGE,MATERNAL DRUG THERAPY PRENATAL TEST AND DIAGNOSIS.pdf
MATERNAL AGE,MATERNAL DRUG THERAPY PRENATAL TEST AND DIAGNOSIS.pdf
 
infertility.pdf
infertility.pdfinfertility.pdf
infertility.pdf
 
Inborn Errors of Metablism.pdf
Inborn Errors of Metablism.pdfInborn Errors of Metablism.pdf
Inborn Errors of Metablism.pdf
 
Huntington Disease and Mental illness.pdf
Huntington Disease and Mental illness.pdfHuntington Disease and Mental illness.pdf
Huntington Disease and Mental illness.pdf
 
Hematological Disorders.pdf
Hematological Disorders.pdfHematological Disorders.pdf
Hematological Disorders.pdf
 
genetic testing inneanates and childern.pdf
genetic testing inneanates and childern.pdfgenetic testing inneanates and childern.pdf
genetic testing inneanates and childern.pdf
 
PRENATAL TESTING AND DIAGNOSIS.pdf
PRENATAL TESTING AND DIAGNOSIS.pdfPRENATAL TESTING AND DIAGNOSIS.pdf
PRENATAL TESTING AND DIAGNOSIS.pdf
 
REVIEW OF CELLULAR DIVISION MITOSIS AND MEIOSIS 2023.pdf
REVIEW OF CELLULAR DIVISION MITOSIS AND MEIOSIS 2023.pdfREVIEW OF CELLULAR DIVISION MITOSIS AND MEIOSIS 2023.pdf
REVIEW OF CELLULAR DIVISION MITOSIS AND MEIOSIS 2023.pdf
 
sex linked inheritance error of transmission.pdf
sex linked inheritance error of transmission.pdfsex linked inheritance error of transmission.pdf
sex linked inheritance error of transmission.pdf
 
spontaneous Abortion.pdf
spontaneous Abortion.pdfspontaneous Abortion.pdf
spontaneous Abortion.pdf
 
error of transmission.pdf
error of transmission.pdferror of transmission.pdf
error of transmission.pdf
 

Recently uploaded

IATP How-to Foreign Travel May 2024.pdff
IATP How-to Foreign Travel May 2024.pdffIATP How-to Foreign Travel May 2024.pdff
IATP How-to Foreign Travel May 2024.pdff
17thcssbs2
 

Recently uploaded (20)

Stl Algorithms in C++ jjjjjjjjjjjjjjjjjj
Stl Algorithms in C++ jjjjjjjjjjjjjjjjjjStl Algorithms in C++ jjjjjjjjjjjjjjjjjj
Stl Algorithms in C++ jjjjjjjjjjjjjjjjjj
 
“O BEIJO” EM ARTE .
“O BEIJO” EM ARTE                       .“O BEIJO” EM ARTE                       .
“O BEIJO” EM ARTE .
 
Pragya Champions Chalice 2024 Prelims & Finals Q/A set, General Quiz
Pragya Champions Chalice 2024 Prelims & Finals Q/A set, General QuizPragya Champions Chalice 2024 Prelims & Finals Q/A set, General Quiz
Pragya Champions Chalice 2024 Prelims & Finals Q/A set, General Quiz
 
Championnat de France de Tennis de table/
Championnat de France de Tennis de table/Championnat de France de Tennis de table/
Championnat de France de Tennis de table/
 
Features of Video Calls in the Discuss Module in Odoo 17
Features of Video Calls in the Discuss Module in Odoo 17Features of Video Calls in the Discuss Module in Odoo 17
Features of Video Calls in the Discuss Module in Odoo 17
 
The Last Leaf, a short story by O. Henry
The Last Leaf, a short story by O. HenryThe Last Leaf, a short story by O. Henry
The Last Leaf, a short story by O. Henry
 
How to Manage Closest Location in Odoo 17 Inventory
How to Manage Closest Location in Odoo 17 InventoryHow to Manage Closest Location in Odoo 17 Inventory
How to Manage Closest Location in Odoo 17 Inventory
 
Word Stress rules esl .pptx
Word Stress rules esl               .pptxWord Stress rules esl               .pptx
Word Stress rules esl .pptx
 
MichaelStarkes_UncutGemsProjectSummary.pdf
MichaelStarkes_UncutGemsProjectSummary.pdfMichaelStarkes_UncutGemsProjectSummary.pdf
MichaelStarkes_UncutGemsProjectSummary.pdf
 
2024_Student Session 2_ Set Plan Preparation.pptx
2024_Student Session 2_ Set Plan Preparation.pptx2024_Student Session 2_ Set Plan Preparation.pptx
2024_Student Session 2_ Set Plan Preparation.pptx
 
INU_CAPSTONEDESIGN_비밀번호486_업로드용 발표자료.pdf
INU_CAPSTONEDESIGN_비밀번호486_업로드용 발표자료.pdfINU_CAPSTONEDESIGN_비밀번호486_업로드용 발표자료.pdf
INU_CAPSTONEDESIGN_비밀번호486_업로드용 발표자료.pdf
 
Dementia (Alzheimer & vasular dementia).
Dementia (Alzheimer & vasular dementia).Dementia (Alzheimer & vasular dementia).
Dementia (Alzheimer & vasular dementia).
 
factors influencing drug absorption-final-2.pptx
factors influencing drug absorption-final-2.pptxfactors influencing drug absorption-final-2.pptx
factors influencing drug absorption-final-2.pptx
 
....................Muslim-Law notes.pdf
....................Muslim-Law notes.pdf....................Muslim-Law notes.pdf
....................Muslim-Law notes.pdf
 
philosophy and it's principles based on the life
philosophy and it's principles based on the lifephilosophy and it's principles based on the life
philosophy and it's principles based on the life
 
Removal Strategy _ FEFO _ Working with Perishable Products in Odoo 17
Removal Strategy _ FEFO _ Working with Perishable Products in Odoo 17Removal Strategy _ FEFO _ Working with Perishable Products in Odoo 17
Removal Strategy _ FEFO _ Working with Perishable Products in Odoo 17
 
BỘ LUYỆN NGHE TIẾNG ANH 8 GLOBAL SUCCESS CẢ NĂM (GỒM 12 UNITS, MỖI UNIT GỒM 3...
BỘ LUYỆN NGHE TIẾNG ANH 8 GLOBAL SUCCESS CẢ NĂM (GỒM 12 UNITS, MỖI UNIT GỒM 3...BỘ LUYỆN NGHE TIẾNG ANH 8 GLOBAL SUCCESS CẢ NĂM (GỒM 12 UNITS, MỖI UNIT GỒM 3...
BỘ LUYỆN NGHE TIẾNG ANH 8 GLOBAL SUCCESS CẢ NĂM (GỒM 12 UNITS, MỖI UNIT GỒM 3...
 
REPRODUCTIVE TOXICITY STUDIE OF MALE AND FEMALEpptx
REPRODUCTIVE TOXICITY  STUDIE OF MALE AND FEMALEpptxREPRODUCTIVE TOXICITY  STUDIE OF MALE AND FEMALEpptx
REPRODUCTIVE TOXICITY STUDIE OF MALE AND FEMALEpptx
 
Exploring Gemini AI and Integration with MuleSoft | MuleSoft Mysore Meetup #45
Exploring Gemini AI and Integration with MuleSoft | MuleSoft Mysore Meetup #45Exploring Gemini AI and Integration with MuleSoft | MuleSoft Mysore Meetup #45
Exploring Gemini AI and Integration with MuleSoft | MuleSoft Mysore Meetup #45
 
IATP How-to Foreign Travel May 2024.pdff
IATP How-to Foreign Travel May 2024.pdffIATP How-to Foreign Travel May 2024.pdff
IATP How-to Foreign Travel May 2024.pdff
 

HUMAN GENOME PROJECT AND GENE THERAPY.pdf

  • 1. GRACIOUS COLLEGE OF NURSING ABHANPUR RAIPUR C.G HUMAN GENOME PROJECT AND GENE THERAPY GENE THERAPY PRESENTED BY OM VERMA ASSISTANT PROFESSOR
  • 2.
  • 3. GENOME Genome is the complete DNA sequence contained in the chromosomes of an organism which carries its complete genetic information. The human genome is a complete set of nucleic acid genome is a complete set of nucleic acid sequences for humans, In Humans, the genome consists of 23 pairs of chromosomes located in the cell's nucleus,
  • 4. Human Genome Project :- It is a mega project was an international scientific research project with the goal of determining the base pairs that make up human DNA, and of identifying, mapping and sequencing all of the genes of HUMAN GENOME PROJECT mapping and sequencing all of the genes of the human genome from both a physical and a functional viewpoint. It started in 1990 and was completed in 2003. 13 Year project. under Dr. james D Watson at the United States National Institute of Health .
  • 5. DEFINITION Human Genome Project was an international research effort to determine the sequence of the human genome and identify the genes that it genome and identify the genes that it contains. According to S.S Randhawa
  • 6. GOALS – 1. Identify all the 20,000 – 25,000 ( approx ) gens in Human DNA. ( counting of alleles low and multiples of alleles and their gens locus ) 2. Determine the sequences of the 3 billion chemical base 2. Determine the sequences of the 3 billion chemical base pairs that make up human DNA. 3. Develop software for large scale DNA analysis ,store all found information in data base and improve tools for data analysis. 4. Collect and distribute .
  • 7. 5. Transfer related technologies to the private sectors . 6. Improve the sequencing technology by developing New and more effective methods 7. Analyze sequence variations in the human genome, such as single nucleotide polymorphisms ( the such as single nucleotide polymorphisms ( the presence of two or more variant forms of a specific DNA sequence ) and other DNA sequence variations’ 8. To map all of human gene
  • 8. 9. To find total number of gene 1o. Study the ethical ,legal and social Issues genetic ( right and wrong ) research that may arise from the project.
  • 9. USES OF HUMAN GENOME – 1. Etiology of cancers, Alzheimer's disease etc. 2. Defining the pathogenesis of a 2. Defining the pathogenesis of a disease and to study the disease process at molecular 3. Sensitivity, of an individual to a variety of illness. level.
  • 10. 4. Precise and accurate, new ways to prevent a Number of diseases that affect human beings. 5. To diagnose and treat disease. Target genes for treatment and management of disease. 6. Human development and anthropology. ( human societies ) 7. Improving knowledge on mutation .
  • 12. INTRODUCTION Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective disease by repairing or reconstructing defective genetic material. healthy gene may replace a damaged (mutated) gene, inactivate a mutated gene or introduce an entirely new gene.
  • 13. Gene therapy is a define as the replacement of a deficiency gene product or repair of abnormal gene within an individual’s cells and tissue to treat disease. tissue to treat disease. According to Siddhartha Mukherjee
  • 14. Gene therapy is a technique which involves the replacement of defective genes with healthy ones in order to treat genetic ones in order to treat genetic disorders. According to AND
  • 15. Gene therapy is the insertion of genes into an individuals cells and tissue to treat disease and hereditary disease and treatment in which the genes that are missing or not normal in cell are are missing or not normal in cell are replace with normal genes that condition is called gene therapy According to V.K. Agarwal
  • 16. Applications of gene therapy: Gene therapy should only be used to treat serious diseases used to treat serious diseases The various applications are:
  • 17. INTRODUCTION OF NORMALLY FUNCTIONING GENE: The most common form of gene therapy involves the insertion of "normal" functional genes into the genome. When there is genes into the genome. When there is defective gene in the cells, it can not produce the respective protein which normal gene produces. In order to produce a protein product, the defective gene causing the disease is replaced by a healthy copy of the gene.
  • 18. INTRODUCTION OF TOXIC GENE: Genes whose products are toxic may be introduced to cause direct death of malignant cells or infective organisms in the cells. INTRODUCTION OF PRODRUG GENE: The products of introduced genes may act as prodrugs and kill the cells by conventionally administered cytotoxic agents. by conventionally administered cytotoxic agents. INTRODUCTION OF ANTIGEN OR CYTOKINE GENE: This may stimulate immune response against neoplastic cells or infective disease and result in immune mediated destruction.
  • 19. INACTIVITY/KNOCKING OUT GENE: The non functional defective gene may be removed by this approach.
  • 21. The cells in the body can be divided into two main categories :- 1. Somatic Cell Therapy 1. Somatic Cell Therapy 2. Germ Cell Therapy
  • 22. 1. SOMATIC CELL THERAPY Somatic cell gene therapy involves the placement of a human gene into a living person's somatic cells—cells that do not produce the eggs and sperm that in turn produce the next generation. Transfer of genes into the somatic cells of the patients as cell of bone marrow Somatic cell gene therapy would cell of bone marrow Somatic cell gene therapy would aim to cure a disease only in the patient, Somatic gene transfer is carried out by two methods, such as 1. IN VIVO ( a process) performed or taking place in a living organism.). 2. EX VIVO (outside the body or in the laboratory).
  • 23. 1. IN VIVO IN VIVO :- In vivo gene therapy, the therapeutic genetic material is introduced directly into the patients body new genes are inserted directly into body. is the insertion of functioning genes directly to the human body through the bloodstream. 2. EX VIVO Ex vivo :- Gene therapy involves removing cells from body, genetically modification ex vivo in a laboratory. , and placing and transfer them back into patient body.
  • 24. 2. GERM CELL THERAPY- Germline gene therapy is when DNA is transferred into the cells that produce reproductive cells, eggs or sperm, in the body. This type of therapy allows for the correction of disease-causing gene variants for the correction of disease-causing gene variants that are certain to be passed down from generation to generation. Germ cell therapy in germ cell are used and it result permanent change that are passed on to the future generations .
  • 25. METHOD OF GENE THERAPY METHOD OF GENE THERAPY
  • 26. VECTORS IN GENE THERAPY The most common form of gene therapy involves of normal gene into the genome with the help of certain carriers called vectors vectors There are two vectors are used in gene therapy 1. VIRAL VECTORS 2. NON VIRAL VECTORS
  • 27. 1.VIRAL VECTORS :- They are the most commonly used vectors. These viruses are genetically modified such that they do not to cause disease, in patients who receive gene therapy. Various patients who receive gene therapy. Various viruses that can be used as vectors for gene therapy include: retroviruses, adenoviruses, herpes simplex viruses.
  • 28. 2. NON VIRAL VECTORS Non viral method: Gene delivery can also be carried by non viral methods. The non- viral vectors are Naked DNA, particle based and chemical based. based and chemical based. Method:- Direct introduction The therapeutic naked DNA is directly introduced into target cells.
  • 29. Liposome mediated DNA transfer : liposome mediated gene delivery is the way of transferring gene into the liposome to mediate gene into the cell and perform its desired function. liposome mediated gene delivery is the way of transferring gene delivery is the way of transferring gene into the liposome to mediate gene into the cell and perform its desired function. Human artificial chromosome Stem cell of bone marrow
  • 30. ADVANTAGE OF GENE THERAPY 1. Eliminate and prevent hereditary disease. 2. It help to eliminate disease from future generation. 3. By this disease therapy possible to cure the heart disease or aids and cancer .