GENE THERAPY-1 : GENE THERAPY-1 DR. SEEMA UPADHYE
Introduction : Introduction In Gene therapy the genes are inserted into a living cell and tissue to treat a disease..
Mainly this therapy is used to treat hereditary diseases.
The defective gene is replaced by a functional gene.
Method : Method Genes were introduced straight into human cells and mainly focused on single gene defects like hemophilia, sickle cell anemia, cystic fibrosis and muscular dystrophy.
This method is quite difficult because to transfer or deliver large sections of DNA and the delivering them to the correct site is also very difficult.
most gene therapy studies today are used for cancer and hereditary genetic diseases .
Types of gene therapy : Types of gene therapy Somatic gene therapy
Germ Line Gene therapy
Somatic gene therapy : Somatic gene therapy Somatic cells as we all understand restricts to individual life. It is not inherited by the patient’s children.
In this technique the therapeutic genes are transferred into the somatic cells of a patient.
Germ line gene therapy : Germ line gene therapy In the case of germ line gene therapy, germ cells, i.e., sperm or eggs, are modified by the introduction of functional genes, which are ordinarily integrated into their genomes. Therefore, the change due to therapy would be heritable and would be passed on to later generations.
This new approach, theoretically, should be highly effective in counteracting genetic disorders and hereditary diseases. However, many jurisdictions prohibit this for application in human beings, at least for the present, for a variety of technical and ethical reasons
Methodology : Methodology 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 a normal gene through homologous recombination.
The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.
The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.
Spindle transfer is used to replace entire mitochondria that carry defective mitochondrial DNA.