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Background
In the 1980s ,human genes to be
sequence and clone (insulin)
On September 1990, the first approved
gene therapy procedur on four-year old
Ashanti DeSilva (SCID=Severe Combined
Immunodeficiency)
Methods
A normal gene may be inserted into a
nonspecific location within the genome to
replace a nonfunctional gene. This
approach is most common
An abnormal gene could be swapped for
normal gene through homologous
recombination
2. Non-viral methods
Naked DNA
Oligodeoxynucleotide
Lipoplexe and polyplexes
3.Hybrid methods
Viruses
All viruses attack their hosts and introduce
their genetic material into the host cell as
part of their replication cycle
This genetic material contains basic
instruction of how to produce more
copies of these viruses.
Molecular biologists realized that viruses
like this could be used as vehicles to carry
good genes into a human cell.
Retrovirus
The Genetic material is in the form RNA
molecules
The Genetic material of their hosts is in the form
DNA
When a retrovirus infects a host cell, it will
introduce its RNA together with some enzymes
into the cell
This RNA molecule from the retrovirus must
produce a DNA copy from its RNA molecule
before it can be considered for part of the
genetic material of the host cell
Adenovirus
The genetic material is in the form ds DNA
Causing respiratory (especially the common
cold), intestinal, and eye infections in humans
When these viruse infect a host cell, they
introduce their DNA molecule into the host.
The genetic material of the adenoviruses is not
incorporated into the host cells genetic material.
The DNA molecule is left free in the nucleus of
the host cell
Non-viral methods
Non-viral methods present certain
advantages over viral methods
simple large scale production
low host immunogenecity
Low levels of transfection and expression of
the gene held non-viral methods at a
disadvantage
Naked DNA
Simplest method of non-viral transfection
Delivery of the naked DNA such as
electroporation and the use of a gene
gun, which shoots DNA coated particles
into the cell using high pressure gas
liposom
e
Hybrid methods
Hybrid methods developed that
combine two or more techniques.
Virosome are one example; they
combine liposomes with an inactivated
HIV or influenza virus.