3. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
4. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
5. Gene Therapy: is the insertion,
alteration, or removal of genes within an
individual's cells and biological tissues
to treat disease.
It is a technique for correcting defective
genes.
Definition of Gene Therapy
6. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
7. Aim of Gene Therapy
The most common form of gene therapy
involves the insertion of functional genes into
an unspecified genomic location in order to
replace a mutated gene, but other forms
involve directly correcting the mutation or
modifying normal gene that
enables a viral infection.
8. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
9. Somatic gene therapy
In Utero gene therapy
Germ line gene therapy
Types of gene therapy
10. In the case of somatic gene therapy, the
therapeutic genes are transferred into
the somatic cells of a patient. Any
modifications and effects will be
restricted to the individual patient only,
and will not be inherited by the patient's
offspring or later generations.
Somatic gene therapy
11. InIn UteroUtero gene therapygene therapy
It involves gene therapy of fetuses
whilst in the womb. There is some
concern that the germline
will be affected.
12. In this case; Germ cells (Ex. sperm or
eggs) are modified by the introduction
of functional genes, which are
integrated into their genomes.
Therefore, the change due to therapy
would be heritable and would be passed
on to later generations.
Germ line gene therapy
13. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
14. General principles of Gene
therapy
Identification and characterization of gene
Cloning of gene
Choice of vector
Method of delivery
Expression of gene
15. Non-pathogeneic.
Capable of integrating DNA into host
genome at a single site.
Able to carry large amounts of DNA.
Unable to cause immune response.
Target cells specifically.
Characteristics of the ideal vector
16. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
17. Viral vectors.
Non-viral delivery.
DNA delivery is mainly
accomplished by either:
25. Particle bombardment. DNA is fired through
the cell walls and membranes on metal
particles. This method was originally
developed to get DNA into plants. However, it
has also been used to make transgenic animals
and is occasionally used for humans.
Particle bombardment
26. Receptor-mediated uptake. DNA
is attached to a protein that is
recognized by a cell surface
receptor. When the protein enters
the cell, the DNA is taken in with
it.
Receptor-mediated uptake
27. Liposomes are spherical vesicles
composed of phospholipid. They
have been used in around 10% of
gene therapy trials.
Liposomes
28. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
32. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
33. 1.1. Short LivedShort Lived
2. Immune Response2. Immune Response
3. Viral Vectors3. Viral Vectors
– may cause disease once insidemay cause disease once inside
Problems with GeneProblems with Gene
TherapyTherapy
34. 4. Multigene Disorders4. Multigene Disorders
– Heart disease, high blood pressure,Heart disease, high blood pressure,
Alzheimer’s, arthritis and diabetesAlzheimer’s, arthritis and diabetes
are hard to treat because you need toare hard to treat because you need to
introduce more than one geneintroduce more than one gene
5. May induce a tumor if integrated in5. May induce a tumor if integrated in
a tumor suppressor gene becausea tumor suppressor gene because
insertional mutagenesisinsertional mutagenesis
Problems with GeneProblems with Gene
TherapyTherapy
35. Definition of Gene Therapy
Aim of Gene Therapy
DNA delivery in Gene Therapy; How & With What
General Principles of Gene Therapy
Gene Therapy Approach
Gene Therapy Problems
Inherited Diseases Treated by Gene Therapy; Trails
Types of Gene Therapy
