Gene Therapy

Gene therapy is the insertion, alteration, or removal of genes within an individual's cells and biological tissues to treat disease. It is a technique for correcting defective genes that are responsible for disease development.


Types of gene therapy


1. Germ line gene therapy :


Germ cells, i.e., sperm or eggs, are modified by the introduction of functional genes, which are integrated into their genomes. Therefore, the change due to therapy would be heritable and would be passed on to later generations.


2.Somatic gene therapy:


The therapeutic genes are transferred into the somatic cells of a patient. Any modifications and effects will be restricted to the individual patient only, and will not be inherited by the patient's offspring or later generations.


Vectors in gene therapy


1. Viruses
Retroviruses
Adenoviruses
Envelope protein pseudotyping of viral vectors
Replication-Competent Vectors
Cis and trans-acting elements
Herpes Simplex Virus

    

2.  Non-viral methods
Injection of Naked DNA

3. Physical Methods to Enhance Delivery

Electroporation

Gene Gun

1. 4.Chemical Methods to Enhance Delivery

 

Magnetofection
Oligonucleotides
Lipoplexes and polyplexes
Dendrimers