Gene Therapy: Challenges and Concerns

Gene therapy is the introduction, removal or change in genetic material (typically means DNA and RNA) in the cells of a patient to treat an inherited or developed disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve human body’s ability to fight disease.

Benefits of Gene Therapy

• Treatment of Rare Diseases
• Therapeutic benefits of Gene Therapy remain effective for a long period of time
• Accuracy

What are the challenges associated with Gene Therapy?

• Complexity of Gene Delivery and Activation.
• It is also crucial to prevent the gene from being introduced into the wrong cells.
• Unwelcome immune response to introduced genes (through vectors) could cause serious illness or even death.
• Vulnerability to disrupt other cells.
• Commercial viability high cost of developing a treatment makes it an unappealing prospect for pharmaceutical companies.

Ethical Concerns related to Gene Therapy

• Safety and unintended consequences are of primary concerns,
• Informed consent,
• Justice and equity,
• Objections to the use of human embryos for genome-editing research.

Suggestions

• Use as Last Resort: Gene Therapy should be utilized only for rare diseases that cause serious illness/fatality, when no other treatment alternatives are available.
• Monitoring: There is a need to have data on the health risks and benefits, as well as the requirement for continuous monitoring throughout clinical trials.
• Regulation and Scrutiny: Gene Therapy experiments and tests must be subjected to strict regulation and scrutiny to keep any unethical activity (like designer babies) under check and prevent commercial misuse.
• IP Rights and Equitable Access: The WHO should work with all stakeholders to encourage relevant patent holders to help ensure equitable access to human genome editing interventions.
• Engagement and Education