Gene Therapy Poised for Mainstream Adoption in Singapore by 2030s

Gene therapy is on the verge of becoming a mainstream treatment option in Singapore by the 2030s, as medical researchers actively explore innovative gene editing techniques for various genetic diseases. Researchers at institutions such as the National University Heart Centre Singapore are conducting trials to address conditions like transthyretin amyloid cardiomyopathy, a rare genetic heart disease with no current cure.

One promising avenue involves gene editing approaches that not only target adult patients but also explore the potential for treating foetuses in utero. If successful, these advancements could revolutionize the treatment landscape for genetic disorders in the coming decade.

Innovative Research and Promising Developments

According to Assistant Professor Lin Weiqin, clinical director of the Heart Failure and Cardiomyopathy Programme at the National University Heart Centre, gene therapy represents a significant breakthrough. Current trials aim to correct genetic defects at their source, and if approved, this could lead to Singapore’s first commercial application of CRISPR-Cas9 technology on human patients.

“It is groundbreaking, it is exciting, and it might pave the way for future gene editing trials for other conditions,” Lin stated. He indicated that conditions such as high cholesterol, hypertension, obesity, and diabetes could potentially be addressed through similar gene therapy strategies. The study is expected to take three to four years to complete, suggesting that widespread adoption of gene therapy could be possible by the 2030s.

CRISPR-Cas9, a powerful DNA editing tool derived from a natural genome-editing system used by bacteria, has already shown promise in treating blood cancers and pediatric neuromuscular diseases. The process involves identifying a specific DNA sequence for alteration, after which a guide RNA directs the Cas9 protein to cut the DNA at the precise location. The cell naturally repairs itself, but scientists can introduce a template DNA sequence to ensure accurate rebuilding, potentially correcting defective genes or inserting new ones.

Addressing Ethical Concerns in Gene Therapy

While the potential of gene therapy is exciting, experts caution that ethical considerations must accompany its development. The Bioethics Advisory Committee (BAC) in Singapore recently outlined guidelines emphasizing the importance of safeguarding ethical standards in gene editing research. At a conference in March 2024, the committee advised that embryos used for research should not develop beyond 14 days, as they begin to acquire a more “human-like” structure at that stage.

“Any potential application to embryos or gametes might be inherited by future generations,” warned Assistant Professor G Owen Schaefer from the National University of Singapore’s Centre for Biomedical Ethics. He stressed the unpredictability of long-term consequences, advocating for reliance on established methods to mitigate the risk of inherited genetic diseases until gene editing technology matures further.

Intrauterine gene therapy also poses risks, as mothers may experience side effects during the treatment of their foetuses. Research in this area is ongoing, and scientists are working diligently to ensure that any approach taken is both safe and effective.

As Singapore moves towards potentially mainstreaming gene therapy, the intersection of innovative medical research and ethical responsibility remains a crucial conversation. The future of genetic disease treatment lies in balancing groundbreaking advancements with the imperative to safeguard human welfare.