Somatic cell gene therapy.

triteritzyBiotechnology

Dec 14, 2012 (4 years and 8 months ago)

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Gene therapy






Gene therapy



Using gene technology to treat genetic disorders



Usually involves adding a functional copy of a gene to
cells which only contain a dysfunctional copy.



The dysfunctional gene is not removed, which means
that this technique works best with recessive disorders
(why?)



Two types,
somatic

cell and
germ line
cell gene
therapy






Somatic cell gene therapy.



Somatic cells are differentiated cells, in which some genes
are transcribed and some are not.



Gene therapy by augmentation


adding new, functional
copies of a dysfunctional gene to the cells that express that
gene.

Eg



the correct copy of the gene that causes cystic fibrosis
can be added to cells in the lining of the lungs using a
liposome or a virus vector.


SCID

S
evere,
C
ombined
I
mmuno
d
eficiency



A condition which leads to complete dysfunction of
the immune system



Various forms, one of which is caused by a recessive
mutation in the gene for the enzyme Adenosine
Deaminase

(ADA), which leads to the build up of toxins
which kill T lymphocytes.

Somatic cell gene therapy in cancer
treatment.



Gene therapy can be used to introduce new genes into
tumour cells.

Germ line cell gene therapy



This involves introducing a new gene into a sperm cell, an ovum,
a zygote or a very early embryonic cell.



This means that the new gene will be seen in all the body cells
of individual that develops from that germ line cell.



That individual will also be able to pass on the modification to
the next generation


This is illegal in humans pretty much everywhere….


Comparison of germ line cell and somatic cell
gene therapy


germ line cell

Somatic cell

Comparison of germ line cell and somatic cell
gene therapy


germ line cell

Somatic cell

Xenotransplantation


Transplantation of cells tissues and organs
between animals of different species


Why would this be potentially useful?


Why is it problematic?


How could genetic engineering make
xenotransplatation

a more realistic option?


Potential Benefits and Risks of Genetic Engineering



Micro
-
organisms



Animals



Plants



Humans