Gene therapy is an attempt to cure hereditary or genetic diseases.
Genes are inserted into a person’s cells and tissue to treat the disease.
The first clinical gene therapy was given in 1990 to a 4-yr old girl with adenosine deaminase (ADA) deficiency.
This enzyme is required for breakdown of deoxyadenosine into uric acids.
In the absence of ADA toxic deoxyadenosine is accumulated and destroy the infection fighting immune cells called T-cells and B-cells.
This disorder is caused due to the deletion of the gene for adenosine deaminase in chromosome 20.
Treatment:
Treated by bone marrow transplantation.
Enzyme replacement therapy, involving repeated injections of the ADA enzyme
Lymphocytes from the blood of the patient are grown in a culture. A functional ADA cDNA is then introduced into these lymphocytes and returned into the body.
The patient required periodic infusion of genetically engineered lymphocytes because these cells are not immortal.
Functional ADA cDNA introduced into cells at early embryonic stages, could be the permanent cure.
Molecular diagnosis:
Early detection of disease is not possible by conventional methods (serum and urine analysis)
Molecular diagnosis techniques:
Recombinant DNA technology.
Polymerase chain reaction (PCR)
Enzyme linked Immuno-sorbent Assay (ELISA)
Very low concentration of a bacteria or virus can be amplified and detected by PCR.
It used to detect genetic disorders.
PCR is use full to mutation in genes in suspected cancerous patient:
A single stranded DNA or RNA tagged with radioactive molecule (probe) is allowed to hybridize to its complementary DNA in a clone of cells followed by detection using autoradiography.
The clone having mutated gene unable make complementary bonding of probe, hence not appears in photographic film.
TRANSGENIC ANIMALS:
Animals that have an alien DNA which able to express in it is called transgenic animals.
Reasons for creation of transgenic animals:
Normal physiology and development:
Transgenic animals are specifically designed to allow study of:
How the genes are regulated.
How the gene affects normal functioning of body
How it affects growth and development. E.g. insulin like growth factor.
The animals made transgenic to know the biological effect and result.
Study of disease:
Transgenic animals are designed to understand how genes contribute to the development of disease like cancers, cystic fibrosis, rheumatoid arthritis and Alzheimer’s.
Biological products:
Transgenic animals are used to produce biological product of human interest:
α-1-antitrypsin used to treat emphysema.
Proteins for treatment for PKU and cystic fibrosis.
Transgenic cow Rosie, produce human protein enriched milk (2.4 gm/lit. human α-lactalbumin)
Vaccine safety:
Transgenic mice are being developed and use in testing the safety of vaccines before they are used for humans.
Polio vaccine is tested in mice.
Chemical safety testing:
This is also known as toxicity/safety testing.
Transgenic animals are made to known the effect of toxic chemicals.
ETHICAL ISSUES:
GEAC (Genetic Engineering Approval Committee) set up by Indian Govt, which will make decisions regarding validity of GM research and safety of introducing GM-organisms for public services.
A patent is the right granted by a government to an inventor to prevent others from commercial use of his invention.
Patents granted for biological entities and for products derived from them; these patents are called biopatents.
27 documented varieties of Basmati are grown in India.
Biopiracy is the term used to refer to the use/exploit or patent, of biological resources by multinational companies and other organizations without proper authorization from the countries and people concerned without compensatory payment.
CBSE Biology (Chapter Wise) Class XII ( By Mr. Hare Krushna Giri )
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