Gene Therapy The deliberate introduction of genetic material into human somatic cells for therapeutic, prophylactic or diagnostic purposes.
Protocols of Gene therapy Somatic versus Germline Gene therapy Ex vivo versus in vivo Gene therapy
Somatic Cell Gene therapy Manipulating the genome of somatic cells in selected tissues .The effect of the therapy are planned to be limited to the individual undergoing treatment and are not designed to introduce potentially heritable genetics modification.
Germline Gene therapy It is possible to permanently modify the germline of animals,which makes the young generation normally .In theory, it should be possible to do in people.
Transfer of the target DNA Physical Method Chemical Method Membrane integration Virus vector Receptor system Homo-recombination
Membrane integration lipisome delivery system A lipisome is an ambipathic molecular capable of binding to DNA and incorporating it into a lipid bilayer which facilitates endocytic entry of the DNA into cells. p346
In vivo Gene therapy Attempt to treat the patient with a gene delivery vehicle that will transfer the target gene to the appropriate target organ and directly modify the genome of the target cells.
Regulation of gene expression— Antisense Technology A method for negating gene expression involves in the introduction of a single strand DNA, RNA or even synthetic nucleic acid derivative that is complementary to the sequence of the target gene. The complementary antisense nucleic acid can then bind to the single mRNA translated from the targeted gene to create a double strand molecule that is no longer able to translate.
DNA Antisense DNA transcription RNA Sense Strand
Stem-cells transplantation Stem cells are unspecialized cells that defined by their capacity for self-renewal and the ability to differentiate into the cell types found in the tissue which they are derived.