THE ASPECTS OF

GENE THERAPY
 INTRODUCTION

Gene therapy is one of the modern attempts to reduce the negative

clinical consequences of genetic diseases. This emerging field needs to
be explored more by researchers and medical practitioners to maximize
it’s enormous potential at eliminating global pathologic conditions that
are not yet curable.
 GENE THERAPY
DEFINED
Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the
biological properties of living cells for therapeutic use.

Gene therapy is a technique that modifies a person’s genes to treat or cure diseases.

Gene therapies can work by several mechanisms:

- Replacing a disease-causing gene with a healthy copy of the gene

- Inactivating a disease-causing gene that is not functioning properly

- Introducing a new or modified gene into the body to help treat a disease
Gene therapy products are being studied to treat diseases
including cancer, genetic disease and infectious diseases.

There are a variety of types of gene therapy products,

including :

- Plasmid DNA
- Viral vectors
- Bacterial vectors
- Human gene editing technology
- Patient-derived cellular gene therapy products
 TWO TYPES OF GENE
THERAPY METHODOLOGY
Ex Vivo
- Gene therapy includes
removing cells from your body,
modifying them and placing
them back to your body.
-Called Ex Vivo because the
cells are treated outside the
body
In Vivo
- New genes are inserted
directly into your body or
genes are changed in cells
when the cells are inside the
body
- Called In Vivo because the
gene is transferred to cells
inside the patient’s body
 BENEFITS OF GENE
THERAPY

Gene therapy involves replacing a faulty gene or adding a new gene in an

attempt to cure disease or improve your body’s ability to fight disease.

Gene therapy has the potential to treat a wide range of diseases, including
cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Gene therapy has the potential to improve or restore function in affected

tissues or cells over time, allowing a patient to manage his or her disease
without the need for ongoing treatments.
 DETRIMENTAL
EFFECTS OF GENE
THERAPY
There are concerns about gene therapy including the procedure’s risk and
could develop an immune reaction to the virus upon receiving the
treatment. The body’s immune system may perceive the newly acquired
viruses as intruders and attack them, which can cause inflammation and in
severe cases, organ failure.

Gene therapy is only a clinical trial. The efficiency of gene transfer and the
expression of the corrective genes in the human patients is still very low,
and it also has a side effect the includes fever, severe chills, blood pressure
drop, nausea, vomiting, headache, serious health risks, such as toxicity,
inflammation, and cancer which can lead to death.