Gene Therapy: Is Still In Its Infancy But The Future Looks Promising

Gene Therapy Leaves a Vicious Cycle..!!

Gene therapy is the introduction of DNA into a patient to treat a genetic disease or a disorder. The newly inserted DNA contains a correcting gene to correct the effects of a disease, causing mutations. Gene therapy is a promising treatment for genetic diseases and also includes cystic fibrosis and muscular dystrophy. Gene therapy is a suitable treatment for infectious diseases, inherited disease and cancer.

Over the last few centuries, infectious diseases have been understood and tackled, through advances in sanitation, anti-microbial medications and vaccination. One day we may also be able to tackle genetic diseases – lifelong conditions arising from mutations that we inherit from our ancestors or that occur during our development.

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The future Of Gene Therapy

We will soon see an increasing number of patients helped by both gene replacement therapy and by CRISPR-mediated gene correction. But the work is likely to be focused on a few specific diseases rather than there being a broad advance across all genetic diseases.

The diseases treated first will share some key characteristics: the genetic defects would be well-understood; they must affect a tissue we can get at easily (blood will be easier than brains and bones); the conditions would be serious and have no other effective treatments.

And they must be so costly in terms of human suffering and economic burdens that a complex and expensive treatment such as gene therapy becomes a viable option.

This means common blood and immune disorders are likely to feature in the first generation trials. Cancer is also a genetic disease, but one typically caused by mutations that accumulate in our cells over time rather than by inherited mutations, and somatic gene therapy, in the form of immunotherapy, involving enhancing the capacity of our immune systems to fight cancer may also become common.

The age of gene therapy is arriving but it will be gradual, not sudden. But incrementally, more people will benefit from these treatments.

In the long term, as we all become aware of mutations we carry in our own genomes that may affect our offspring, there may be pressure to correct more and more genetic lesions. This will remain too risky and expensive for many years so gene therapy will likely remain a niche and specialist treatment for the foreseeable future.

Dominated Companies Working on Gene Therapy

Sangamo Therapeutics, bluebird bio, uniQure N.V., AveXis, Vineti, Solid Biosciences., Spark Therapeutics, CHIMERON BIO, RENOVA THERAPEUTICS, HORAMA S.A.

For More Details Of Gene Therapy Industry Analysis By Cell Type (Somatic Gene Therapy, Germline Gene Therapy); By Application (Genetic Disorder, Cancer, Neurological Disorder, and Others) Key Segmentation, Geographical Supply And Demand, Worldwide Manufacturers

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References:

  • theconversation.com
  • theinsightpartners.com