GENE

THERAPY
BY: Kirsten Nicole C. Ledesma
01 What is Gene Therapy?
02 History of Gene Therapy
TABLE OF
03 Pros and Cons of Gene Therapy CONTENTS
04 Types of Gene therapy
05 Vectors in Gene therapy
 Gene therapy is a medical field which
focuses on the genetic modification
of cells to cure rare diseases and
cancer
WHAT IS GENE
It is done by repairing or
THERAPY? reconstructing defective genetic
material

3
 A BRIEF HISTORY OF GENE THERAPY
Gene Therapy was first introduced in the year 1928
when men where still not very versed in biotechnology.
It was sir. Frederick Griffith who described the
principles behind this

The first approved genetic therapy clinical research in the US took place on 14 September
1990, at the National Institutes of Health or the NIH. It was after French Anderson, MD, was
“dubbed as 'the father of therapy' after a team he led in 1990 cured a 4 year old girl named
Ashanti DeSilva of a very severe genetic defect.

The first genetic therapy however, was licensed in China in 2003. Designed for the
treatment of neck and head cancer. Despite the good this brings, It did not make it to other
countries. It has yet to be started world wide.
THE PROS AND CONS OF
GENETIC THERAPY
Pro’s Con’s

- Can/it cures cancer & rare diseases - Incredibly risky

- improves body’s immune system - Expensive
- A very promising treatment for - Might lead to DNA complications and
diseases harmful mutations
- Offers the possibility of a permanent - Can cause genetic disorders and
cure long term complications
- Experimental
TYPES OF GENE
THERAPY

Germline cell therapy fixes/corrects genetic defects in Somatic cell therapy prevents genetic defects in the
the offspring. It transfers its section of DNA to any cell that offspring. It transfers its section of DNA to any cell that
produces eggs or sperm doesn’t produce eggs or sperm
VECTORS OF GENE
THERAPY
Viruses are usually used as vectors as they deliver the new gene by
infecting the cell of the host. There are different types of viruses modified
with their own strengths and weaknesses. Each has different applications
for which they are best suited for.

More examples are retrovirus, adenovirus, and adeno-associated virus.