Case study:Gene Therapy

What is Gene theraphy

• Gene therapy is an experimental technique that uses genes to treat or
prevent disease. In the future, this technique may allow doctors to
treat a disorder by inserting a gene into a patient’s cells instead of
using drugs or surgery.
Cell types
Somatic
• 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, in
which therapeutic DNA (either integrated in the genome or as an
external episome or plasmid) is used to treat disease.
Germline
• In germline gene therapy (GGT), germ cells (sperm or egg cells) 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. Australia, Canada, Germany, Israel, Switzerland, and
the Netherlandsprohibit GGT for application in human beings, for
technical and ethical reasons, including insufficient knowledge about
possible risks to future generations.
HISTORY OF GENE THERAPY
• The first approved gene therapy clinical research in the US took place
on 14 September 1990, at the National Institutes of Health (NIH),
under the direction of William French Anderson
How can Gene therapy benefits us
• Gene therapy replaces a faulty gene or adds a new gene in an attempt
to cure disease or improve your body's ability to fight disease. Gene
therapy holds promise for treating a wide range of diseases, such as
cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
How can Gene therapy affect us Negatively
• Some of these risks may include: The immune system may respond to
the working gene copy that has been inserted by causing
inflammation. The working gene might be slotted into the wrong
spot. The working gene might produce too much of the missing
enzyme or protein, causing other health problems.
How much Gene therapy
• The cost of these treatments, though, ranges from about $500,000 to
$1.5m. And over a lifetime, drugs like nusinersen can be even more
expensive: $750,000 in the first year followed by $375,000 a year
after that – for life. As these prices suggest, it's expensive to get a
gene therapy drug to the market.