The Promise and Challenges of Gene Therapy

The treatment that is surrounded by a lot of debate is the gene therapy which is regarded as an enormous innovation in the treatment of genetic disorders and diseases. As a form of treatment, gene therapy looks to alter the DNA present in a patient’s cell directly, targeting specific genes that are regarded as a disease-generating pathway. Coined as this, this technique satisfies medical science when it comes to incurable diseases such as various inherited diseases, cancers, and viral infections.

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The delivery of gene therapy has been most successful in the treatment of severe combined immunodeficiency or SCID, also known as “bubble boy disease”. Children with SCID are born with a missing immune system, something Gene therapy has managed to rectify by directly installing functional genes to improve the functionality of the immune system amongst the patients.

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However, gene therapy is not without hitches. Transfecting therapeutic genes into the target cell still poses the major issue because circulation of viral vectors that deliver therapeutic genes often poses a risk of immune response or insertional mutagenesis, which has a propensity to lead to cancer. Also gene treatments are very expensive and most patients can barely afford them which is questionable in terms of ethics and economy.

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However, there are challenges such as the lack of effective delivery system, gene regulation, genotoxicity problems, and immune responses, nevertheless, constant innovation and development are ensuring that gene therapy is becoming safer and more efficient. New developments including the CRIPSR-Cas9 along with other similar technology has now expanded the wizardry of genetic manipulations and the future looks bright. As we continue to face these challenges, gene therapy will go on to act as one of the last legs of making personalized medicine a reality of staggering proportions, now providing cures to hitherto incurable diseases and yet evolving as a ground breaking means of bringing about a sea change in healthcare.

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