Summary
At Save Sight Now Europe, we are committed to accelerating the development of treatments for Usher Syndrome Type 1B. We are proud to support a newly funded research program led by Prof. Elvir Becirovic at the University of Zurich. His team is exploring a highly promising dual approach to gene therapy—combining MYO7A gene supplementation with CRISPR-based gene activation—to pave the way for future USH1B treatments. This preclinical work, carried out in advanced animal models, marks an exciting step toward therapies that could transform the lives of those affected by USH1B.
Save Sight Now Europe is proud to fund a cutting-edge research program led by Prof. Elvir Becirovic and his team at the University of Zurich.
Dr. Becirovic work focuses on developing gene therapy strategies for Usher Syndrome Type 1B (USH1B) — the most severe form of Usher Syndrome, which causes profound congenital deafness and progressive childhood blindness due to Retinitis Pigmentosa.
Building on key breakthroughs in mouse models, Prof. Becirovic’s lab is now advancing two complementary approaches:
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Gene Supplementation: The team is using a novel dual AAV vector system based on mRNA trans-splicing to deliver the full MYO7A gene, which is mutated in USH1B.
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Gene Activation: They are also applying CRISPRa (CRISPR activation) technology to epigenetically activate MYO7B — a functionally related gene — as a potential therapeutic alternative.
To ensure the relevance of these approaches for human therapy, the team will conduct preclinical studies in wild-type minipigs. These animals have a retinal structure and eye size similar to humans, allowing for more accurate and meaningful evaluation.
Our support will enable Prof. Becirovic’s team to carry out these critical studies and prepare for future trials using USH1B-specific minipig models. This work brings us closer to safe, effective treatments for children affected by this devastating condition.
This program highlights not only the promise of gene therapy innovation but also the power of collaboration between nonprofit organizations and academic research to accelerate progress for rare diseases like Usher Syndrome.
We’re excited to follow the developments of Prof. Becirovic’s team and look forward to sharing updates as the project moves forward.