Synthesis of amino acids and proteins and many essential biochemical’s which are required for normal functioning of our body is regulated by DNA in our body , which consists of specific genes over them which are responsible for coding for biochemical’s proteins responsible for normal functioning of our body.
Whenever there is change in the genes which can code for proteins and enzymes which are responsible for normal functioning, instead they code for something abnormal proteins, such a change in genes are called as mutant gene.
Mutation in gene may be harmful, may develop in to cancer or may develop in to a disease which affect individual’s life. Gene therapy has a great potential to provide correction in such mutations in genes, a copy of correct gene can be synthesized in laboratory and inserted in to individuals DNA. There are various techniques available to inert a copy of a correct gene in to patient which can be designed either function as to synthesize a deprived biochemical’s and proteins or can be designed to code for biochemical’s and proteins which act as a drug molecule to correct or to mitigate disease condition.
Following are some diseases which are treated successfully in clinical trials:
Leber's congeneital amaurosis (LCA)
Which and genetic disorder developed when both parents have same genetic defect ,( autosomal recessive) which develop in to photoreceptor cells which are abnormally developed, uncontrolled movement of eyes, patient may develop no pupillary reflex and blindness.
This disease can be successfully treated with gene therapy.
Severe combined immunodeficiency (SCID):
Patient suffering from this disease has his immune system defective and has very poor immunity therefore patient is susceptible to infections, even from normal human bacterial intestinal flora. Some patients were required to be kept in to sterile environment.
Adrenoleukodystrophy : Inherited disorder which lead to brain damage .
Parkinson's disease : Parkinson’s disease is caused by damage and death of cells in brain which produces neurotransmitter dopamine.Disease cause serious loss of cognitive abilities , rigidity in movement and shaking, dementia (loss of memory) .
Gene therapy in HIV infection treatment:
HIV virus uses C-C chemokine receptor type 5 a protein which is used by HIV virus as chemical signal for locating T cell for attaching and infecting, it is important factor in process of HIV infection , Researchers are studying how this C-C chemokine receptor type( 5 CCR5) can be knocked off and replaced it with CCR5 delta 32 gene, as it is found that individuals with CCR5 delta 32 are resistant to HIV infection.
Gene therapy research is being focused to provide promising treatments for diseases which are caused due to alteration or mutation in single gene for example for treating sickle cell anemia, muscular dystrophy and cystic fibrosis and haemophilia.
Gene therapy may include techniques such as gene targeting which removes unwanted gene by using enzymes which cut off genes (knock off technique) at peculiar points and insert new correct gene. Enzymes used for specific knock off are called as nucleases, TAL effectors.
A specific carrier for delivering a gene in to cells is require, methods used for delivering a gene in to host cells are as follows,
1) Recombinant viruses
2) Nano particles derived from nanotechnology
4) Gene gun
5) Electroporation, or electropermeabilization
6) Nano Liposomes
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