Innovative Gene-Editing 'Molecular Scissors' Offer New Hope for Chronic Hepatitis B Treatment

Recent advancements in gene-editing technology have introduced a promising approach to combating chronic hepatitis B (HBV), a persistent viral infection that affects millions worldwide, especially in regions like South Africa. Researchers led by Dr. Tiffany Smith of the Antiviral Gene Therapy Research Unit have developed a novel method utilizing TALENs (transcription activator-like effector nucleases) to precisely target and disable the virus's genetic material.

Unlike other gene-editing tools such as CRISPR, TALENs do not rely on guide RNAs, making them effective even in complex DNA structures. The team's breakthrough centers on targeting covalently closed circular DNA (cccDNA), the 'mini chromosome' that HBV uses to hide within liver cells and sustain infection. Current treatments can suppress virus activity but cannot eradicate cccDNA, leading to potential relapse after therapy cessation.

Preclinical studies have shown significant results: in cultured human liver cells, the treatment reduced hepatitis B markers by 80%, and in infected mice, a single dose caused a 99% reduction in circulating viral DNA. Importantly, the therapy was well tolerated, with only mild, temporary inflammation observed.

This innovation could pave the way for more effective, possibly curative, treatments for HBV. The team is combining TALENs with mRNA delivery via lipid nanoparticles, a platform previously successful in vaccine development, to enhance precision and safety. While further research is needed to confirm these findings in humans, this development marks a significant step toward a functional cure for hepatitis B.

Hepatitis B remains a major health concern, especially in resource-limited settings where lifelong antiviral therapy is burdensome. The potential to directly eliminate the virus's genetic blueprint offers hope for a future where hepatitis B can be effectively controlled or eradicated.

Source: https://medicalxpress.com/news/2025-08-generation-molecular-scissors-chronic-hepatitis.html