GENE THERAPY Flashcards
(17 cards)
is a medical approach that aims to treat or prevent diseases by correcting underlying genetic problems, often by introducing a new gene into cells, or by
replacing a faulty gene with a healthy one.
Gene therapy
TYPES OF GENE THERAPY
SOMATIC GENE THERAPY
GERM LINE GENE THERAPY
*It is the transfer of a section of DNA to
somatic cells. Its aftereffects will not be
transmitted on the offsprings thus its
effects are short-lived.
SOMATIC GENE THERAPY
*This can be further SOMATIC GENE THERAPY categorized into two (2):
1.Ex-vivo
2.In-vivo
where cell are manipulated outside the body and then transplanted again onto the body.
1.Ex-vivo
genes are modified in cells still inside the body.
2.In-vivo
*The functional genes , which are to be integrated into the genomes, are inserted in the germs cells.
GERM LINE GENE THERAPY
GENE THERAPY STRATEGIES
GENE AUGMENTATION THERAPY
TARGETED KILLING OF SPECIFIC CELLS
TARGETED INHIBITION OF GENE THERAPY
TARGETED GENE MUTATION CORRECTION
simple addition of functional alleles is used to treat inherited disorders caused by genetic deficiency
of a gene product.
GENE AUGMENTATION THERAPY
It involves utilizing genes encoding toxic
compounds(suicide genes), or prodrugs(reagents which confer sensitivity to subsequent treatment with a drug) to kill the transfected/transformed cells.
TARGETED KILLING OF SPECIFIC CELLS
This is to block the expression of any
diseased gene or a new gene expressing
a protein which is harmful for a cell.
TARGETED INHIBITION OF GENE THERAPY
It is used to correct a defective gene to
restore its function which can be done
at genetic level by homologous
recombination or at mRNA level by
using therapeutic ribozymes or
therapeutic RNA editing.
TARGETED GENE MUTATION CORRECTION
GENE THERAPY APPROACHES
CLASSIC GENE THERAPY
NON-CLASSIC GENE THERAPY
It involves therapeutic gene delivery and
their optimum expression once inside the
target cell. The foreign genes carry out
following function.
CLASSIC GENE THERAPY
It involves the inhibition of expression of
genes associated with the pathogenesis, or
to correct a genetic defect and restore
the normal gene expression.
NON-CLASSIC GENE THERAPY
Target specific types of disease-causing cells.
Therapeutic protein is continuously produced inside thbody, which also reduces the long-term cost of treatment.
Can be manipulated so that it can’t replicate and destroy cells.
Gene expression can be controlled.
ADVANTAGES OF GENE THERAPY
Viral vectors raise safety concerns
Can cause immune response in patients.
Can carry limited amounts of genetic material. Some genes may be too big to fit into some virus.
Expensive for third world countries.
DISADVANTAGES OF GENE THERAPY
