BASICS OF GENE THERAPY INTRODUCTION,HISTORY,TYPES, USES, ADVANTAGES, DISADVANTAGES, DRAWBACKS, GENETIC ENGINEERING,REGULATION

1. SEMINAR PRESENTATION
BY
MIKA GHOSH
B.SC BIOTECHOLOGY (UG-1)

2. TOPIC
GENE
THERAPY

3. Gene therapy is the application of genetic
engineering techniques to alter or replace defective
genes.
INTRODUCTION

5. Defective
gene
Incorrect sequence
of bases
Inability of a gene to code for
the expression of a particular
polypeptide

6.  It was conceptualized around 1970s.
 In 1972 Friedmann and Roblin authored paper in
Science titled ‘Gene therapy for human genetics’
RICHARD ROBLIN
THEODORE FRIEDMANN

7. The first attempt, an
unsuccessful one at gene
therapy treating against
beta-thalassemia was
performed by Martin Cline
on 10 July,1980.
Martin Cline

8. After extensive research on animals throughout the
1980s and a 1989bacterial gene tagging trial on
humans the first success of gene therapy was
demonstrated on 14 Sep 1990 when Ashi Desilva a 4 yrs
old girl was treated for ADA-SCID by French
Anderson.
French
Anderson.

9. 1972 conceptualized
1980 Retrovirus as vector
1990 NIH approval
1993 cancer gene therapy
2002 treatments to sickle cell ,thalassaemia, cystic fibrosis n
several cancers.
2003 genes into brains using liposomes coated in
PEG.
2006 Treatment to X-linked chronic diseases,
prevent immune rejection to, gene based
immune therapy etc organ transplantation.
2007-15 several trials on retinal n congenital
diseases cure any many other diseases.
STRIMVELIS named gene
therapy approved medicine
agency.

10. GENE
THERAPY
SOMATIC
IN-VIVO
EX - VIVO
GERMLINE

11. In somatic cell gene therapy (SCGT), the therapeutic
genes are transferred into any cell other than
a gamete, germ cell, gametocyte or
undifferentiated stem cell.
Any such modifications affect the individual patient
only, and are not inherited by offspring.
Somatic gene therapy represents mainstream basic
and clinical research.

13. EX VIVO
Cells are modified
outside the body &
then transplanted
back in body.
Called ex vivo
because the cells
are treated outside
the body.
IN VIVO
Genes are changed
in cells when the
cells are still in the
body.
Called in vivo
because gene is
transferred inside
the body directly.

15. In Germline gene therapy (GGT), germ
cells (sperm or eggs) are modified by the introduction
of functional genes into their genomes.
Modifying a germ cell causes all the organism's cells
to contain the modified gene. The change is
therefore heritable and passed on to later generations.

17. VECTORS
VIRAL
NON
VIRAL

18. VIRAL NON VIRAL
Introduce their genetic material
into the host cell, tricking the host's
cellular machinery into using it as
blueprints for viral proteins.
DNA VIRUSES
-Adenovirus
- Adeno-associatd virus etc
RNA VIRUS
-Retrovirus
- Lenti virus
Advantages over viral methods,
such as large scale production and
low host immunogenicity.
Naked DNA transfer
Electroportation
Gene gun
Micro injection
Use of oligonucleotides,
Inorganic nanoparticles.

19. FERTILITY
Spermatogenical stem cells from another organism were
transplanted into the testes of an infertile male mouse. The stem
cells re-established spermatogenesis and fertility.
GENE DOPING
Athletes might adopt gene therapy technologies to improve their
performance.

20. Genetic engineering
Genetic engineering could be used to change physical
appearance, metabolism, and even improve physical
capabilities and mental faculties such
as memory and intelligence. For parents, genetic
engineering could be seen as another child
enhancement technique to add to diet, exercise,
education, training, cosmetics and plastic surgery.

21. APPROACHES
GENE
TRANSPLANTATION
GENE
CORRECTION
GENE
AGUMENTATION
•To revert specific mutation in gene of
interest
•To enhance expression of gene interest
GENE ABALATION •Targeted inhibition of gene expression
TARGETED KILLING
OF SPECIFIC CELLS
•By introducing killer gene
•To patient with gene deletion

22. Drawbacks
• Short-lived nature- therapeutic DNA into the genome and the
rapidly dividing nature of many cells prevent it from achieving
long-term benefits. Patients require multiple treatments.
• Immune response - The immune system's enhanced response to
viruses that it has seen before reduces the effectiveness to repeated
treatments.
• Viral vectors- carry the risks of toxicity, inflammatory responses, and
gene control and targeting issues.
• Costly

23. REGULATION
• The Statement on Gene Therapy Research initiated by
the Human Genome Organization(HUGO) in 2001 provides a
legal baseline for all countries emphasizeing human freedom
and adherence to human rights, and offers recommendations
for somatic gene therapy,
• This subject is governed by overlapping regulations from
local and federal agencies, including the Department of
Health and Human Services, the FDA and NIH's Recombinant
DNA Advisory Committee.

24. • NIH serves as the main gene therapy regulator for federally funded
research
 provides funding for research
 maintains a mandatory registry of human genetic engineering
research protocols that includes all federally funded projects.
 An NIH advisory committee published a set of guidelines on gene
manipulation discussing lab safety as well as human test subjects
and various experimental types that involve genetic changes.
• FDA regulates the quality and safety of gene therapy products and
supervises how these products are used clinically.