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What is Genetics?

Genetics is the scientific study about the ways characteristics of living things such as eye color or nose shapes or genetic conditions are passed from one generation to the next. The basic units of heredity are genes; diseases caused by abnormal gene forms are called genetic disorders.

Genomics is the study of all of a person’s genetic material, the interactions of that genetic material, the interactions of that genetic material with the environment, and the subsequent phenotypic changes.
Genetics uses information from one or two genes to explain a disease or condition, whereas genomics examines all of the genetic information to determine biological markers predisposing an individual to disease.

What is Gene Therapy?

Article from University of Utah

Gene therapy could be a way to fix a genetic problem at its source. By adding a corrected copy of a defective gene, gene therapy promises to help diseased tissues and organs work properly. This approach is different from traditional drug-based approaches, which may treat symptoms but not the underlying genetic problems.

Most commonly, gene therapy uses a vector, typically a virus, to deliver a gene to the cells where it's needed. Once it's inside, the cell's gene-reading machinery uses the information in the gene to build RNA and protein molecules. The proteins (or RNA) can then carry out their job in the cells.

But gene therapy is not a molecular bandage that will automatically fix any genetic problem. While many disorders or medical conditions can potentially be treated using gene therapy, others are not suitable for this approach. So what makes a condition a good candidate for gene therapy?


Types of gene therapy

Gene therapy may be classified into the two following types, only one of which has been used in humans:

Somatic gene therapy

As the name suggests, in somatic gene therapy, the therapeutic genes are transferred into the somatic cells (non sex-cells), or body, 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. Somatic gene therapy represents the mainstream line of current basic and clinical research, where the therapeutic DNA transgene (either integrated in the genome or as an external episome or plasmid) is used to treat a disease in an individual.

Several somatic cell gene transfer experiments are currently in clinical trials with varied success. Over 600 clinical trials utilizing somatic cell therapy are underway in the United States. Most of these trials focus on treating severe genetic disorders, including immunodeficiencies, haemophilia, thalassaemia, and cystic fibrosis. These disorders are good candidates for somatic cell therapy because they are caused by single gene defects. While somatic cell therapy is promising for treatment, a complete correction of a genetic disorder or the replacement of multiple genes in somatic cells is not yet possible. Only a few of the many clinical tries are in the advanced stages

Germline gene therapy

In germline gene therapy, germ cells (sperm or eggs) are modified by the introduction of functional genes, which are integrated into their genomes. Germ cells will combine to form a zygote which will divide to produce all the other cells in an organism and therefore if a germ cell is genetically modified then all the cells in the organism will contain the modified gene. This would allow the therapy to be heritable and passed on to later generations.


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