Gene Therapy - Advanced Treatments for a New Era

Published on 06 Jun, 2016

Gene Therapy

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Gene therapy involves inserting/deleting/correcting genetic material into human cells to fight or prevent diseases. It is a promising tool not only for cancer but for several other diseases, such as Parkinson's, HIV, severe combined immuno-deficiencies and hemophilia, to name a few.

In this report, we’ll cover the challenges associated with gene therapy, various modes of administration of gene based therapies, expected future of gene therapy, companies that are investing heavily in gene therapy research, as well as FDA approved gene-based drugs that are currently available in the market or undergoing clinical trials.


Considerable research has been conducted in genomics in the past two decades. Extensive research in the gene therapy domain began in 2001, when two separate versions of human genome sequences were published. These drafts contained 30,000 genes, which were used to decipher gene function, gene abnormality, and malignant alternations at the gene levels.

James Watson was quoted as stating, “We used to think that our fate was in our stars, but now we know, in large measures, our fate is in our genes.” Genes are the functional unit of heredity. When altered, the proteins that they encode are unable to carry out their normal functions. Gene therapy (the use of genes as medicine) is basically used to correct defective genes.

Gene therapy initially encountered a lot of problems as people considered it unethical to use humans as subjects for clinical trials. However, as time passed, gene therapy has proved to be a useful tool to cure several forms of disease.


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