Human beings from more than 5000 different diseases caused by single gene mutations. e.g cystic fibrosis acatalasia Huntingtons chorea. Tay sach's disease Lesch-Nyhan syndrome sickle cell anemia, mitral stenosis, Hunter's syndrome haemophilia several forms of muscular dystrophy etc. In addition many common disorder like cancer, hypertension, atherosclerosis and mental illness seem to have genetic components.
Gene therapy may be defined in broad general terms as follows. Introduction of a normal functional gene into cells which contains the defective allele of concerned gene with the objective of correcting a genetic disorder or an acquired disorder. Application of gene therapy involves the following basic development in genetics molecular biology and biotechnology (1) determination of the role its products in health and disease (2) identification of the gene that play the key role in the development of genetic disorder (3) development of an approach for gene therapy.
The candidate disorder for gene therapy are selected on the basis of this following criteria (1) the disease should be life- threatening (2) gene responsible for the disease has been cloned (3) a precise regulation of the gene should not be required and (4) a suitable delivery system should be available.
Gene therapy may be defined in broad general terms as follows. Introduction of a normal functional gene into cells which contains the defective allele of concerned gene with the objective of correcting a genetic disorder or an acquired disorder. Application of gene therapy involves the following basic development in genetics molecular biology and biotechnology (1) determination of the role its products in health and disease (2) identification of the gene that play the key role in the development of genetic disorder (3) development of an approach for gene therapy.
The candidate disorder for gene therapy are selected on the basis of this following criteria (1) the disease should be life- threatening (2) gene responsible for the disease has been cloned (3) a precise regulation of the gene should not be required and (4) a suitable delivery system should be available.
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