Transcript Clinical application of ribozymes and antisnse oligonucleotide

Gene Therapy
Antisense Technologies
antisense oligonucleotides(ODNs)
 Ribozymes
 DNAzymes
 RNA interference(RNAi).

Gene Therapy...
----idea is to replace defective genes
Gene therapy is a technique for correcting
defective genes responsible for disease
development.
Several approaches for correcting faulty genes
1.
A normal gene may be inserted into a nonspecific
location within the genome to replace a nonfunctional
gene.
2.
An abnormal gene could be swapped for a normal gene
through homologous recombination.
3.
The abnormal gene could be repaired through selective
reverse mutation, which returns the gene to its normal
function.
4.
The regulation (the degree to which a gene is turned on
or off) of a particular gene could be altered.
Deliver the therapeutic gene
Some of the different types of viruses
used as gene therapy vectors:
Retroviruses
 Adenoviruses
 Adeno-associated viruses
 Herpes simplex viruses

Deliver the therapeutic gene
Adenoviruses
Challenge
Efficient delivery
 Enhanced stability
 Minimization of off-target effects
 Identification of sensitive sites in the
target RNAs

Status of gene therapy

The Food and Drug Administration (FDA)
has not yet approved any human gene
therapy product for sale.
Gene Therapy in the clinic
Trials Wordldwide
Phase I
Phase I-II
Phase II
Phase II-III
Phase III
66% Phase I
21% Phase I-II
11% Phase II
0.8% Phase II-III
0.7% Phase III
in spite of 13 year- research only less than 1% of the trials has reached
phase III
For more details

Genetic Modification Clinical Research
Information System (GeMCRIS)
http://www.gemcris.od.nih.gov/
Thanks~!!
~*_*~