By

Jyothi kiranmai .G
(M.Pharm Pharmacology)
MALLA REDDY COLLEGE OF
PHARMACY
 Introduction
 Various approaches
 Types of gene delivery
 Gene therapy of various diseases
 conclusion
 Gene ??
  According to the official Guidelines
for Human Gene Nomenclature, a gene
is defined as "a DNA segment that
contributes to phenotype/function.

1969 - The first single gene is isolated.

1970 - The first artificial gene is made.
Definition: Gene delivery, the insertion of genes (e.g. via retroviral vectors) into
selected cells in the body in order to: 

- Cause those cells to produce specific therapeutic agents. 

- Cause those cells to become (more) susceptible to a conventional therapeutic agent

that previously was ineffective against that particular condition/disease. 

- Cause those cells to become less susceptible to a conventional therapeutic agent. 

- Counter the effects of abnormal (damaged) tumour suppressor genes via insertion
of normal tumour suppressor genes. 

- Introduce other therapeutics into cells.

 Gene modification
 Gene transfer
 Gene transfer in specific cell line
 Replacement therapy

 Corrective gene therapy

 Physical
 Chemical
 Biological
 Somatic

 Germ line
 Retroviruses
 Adenoviruses
 Adeno-associated viruses
 Herpes Simplex Virus
 Alpha virus
 Gene gun
 Liposome mediated gene delivery
 Polymer based gene delivery
 Electroporation
 Proton sponge
A mixture of
polycataionic lipid
and neutral lipid
will result in
formation of
unilamellar
liposome vesicles
that have a net
positive charge due
to positive amine
groups on these
molecules.
 ADA: The First Gene Therapy Trial

A four-year old girl became the first gene therapy patient
on September 14, 1990 at the NIH Clinical Center. She has
adenosine deaminase (ADA) deficiency, a genetic disease
which leaves her defenseless against infections. WHITE
BLOOD CELLS were taken from her, and the normal
genes for making adenosine deaminase were inserted into
them. The corrected cells were reinjected into her. Dr. W.
French Anderson helped develop this landmark clinical
trial when he worked at the National Heart, Lung, and
Blood Institute.
 1985
 
 
 

 The laboratories of Drs. W. French Anderson and Michael

Blaese in the National Heart, Lung, and Blood Institute and the
National Cancer Institute worked together to show
that cells from patients with ADA deficiency can be corrected
in tissue culture . They used a retrovirusto carry the correct
human ADA gene to the cells. Here, Drs. Kenneth Culver (then
of the NHLBI), W. French Anderson, and Michael Blaese pose
with gene therapy patients.
 Gene therapy for insulin dependent diabetis melitus  
 Gene therapy for hemophilia B novel self complementary AAV
vector encoding a codon-optimised human F IX transgene
(scAAV2/8-LP1-hFIXco) into adult subjects with severe HB
 Gene therapy for cystic fibrosis The most recently reported trial
involved eight cystic fibrosis subjects receiving pulmonary
delivery of GL-67 proportions 1:2:0.05) complexed to 7.9–21.1
mg of CFTR plasmid DNA [88].

 Gene therapy for cardiovascular diseases.

 Gene therapy for nervous system disorders.
 Results of world's first gene therapy for inherited
blindness show sight improvement. 28 April 2008. UK
researchers from the UCL Institute of Ophthalmology and
Moorfields Eye Hospital NIHR Biomedical Research
Centre have announced results from the world’s first
clinical trial to test a revolutionary gene therapy treatment
for a type of inherited blindness. The results, published
today in the New England Journal of Medicine, show that
the experimental treatment is safe and can improve sight.
The findings are a landmark for gene therapy technology
and could have a significant impact on future treatments
for eye disease.
 Genes carried in nanoparticles are used to treat
cancer in mice
 Gene therapy cured inheritance blindness in
humans.
 Gene therapy was successful in treating
adenosine deaminase deficiency an auto immune
dis.order in humans.
 Therapeutic gene added to the cell is functionally
active for only short span time.
 Vectors or carriers of therapeutic gene cause
problems.
 Expensive therapy
 Almost every therapy which intended were
succeeded in cell and animal models.
 Its mark in treating humans is most awaiting one
as all are in clinical trials…
 Lets hope for a better way for treating diseases
with ethical values..
 Pharmaceutical biotechnology S.P.Vyas and V.K.
Dixit.
 www.genetherapy.net
 www.nature.com
 www.genomics.energy.gov