Special Interview with Prof. Daniel Scherman

On June 27, 2026, the Editorial Office of Journal of Translational Genetics and Genomics interviewed Prof. Daniel Scherman, Laboratory of Chemical and Biological Technology for Health, Pharmacy Faculty, Paris University, Paris, France.

In his interview, Prof. Scherman outlined the major challenges in translating rare disease research into clinical practice, emphasizing that progress depends on advances across genetics, delivery technologies, clinical development, and international collaboration. He noted that one key bottleneck is the interpretation of variants of unknown significance, which will require functional studies and artificial intelligence to improve genetic diagnosis. He also highlighted the limitations of current delivery systems, including the immunogenicity of viral vectors and the difficulty of delivering genetic medicines to organs such as the brain and lungs. Beyond technological barriers, he stressed the importance of international patient registries and natural history studies for ultra-rare diseases, as well as sustainable funding models to support therapies for very small patient populations. Despite these challenges, he identified rapid progress in RNA therapeutics and CRISPR-based genome editing as particularly promising directions for the future.

Discussing RNA-based therapeutics, Prof. Scherman emphasized that delivery efficiency remains the primary obstacle to broader clinical application, especially beyond the liver, where current technologies have already achieved considerable success. He explained that improving tissue-specific delivery, particularly to muscle and the central nervous system, is essential for increasing the efficacy of antisense oligonucleotides and siRNA therapies. At the same time, he highlighted the expanding therapeutic versatility of RNA medicines, including splice correction, allele-specific silencing, and emerging RNA approaches that enhance translation from healthy alleles. These innovations, together with advances in mRNA-based genome editing, offer new strategies for treating haploinsufficient and other genetic disorders, significantly expanding the therapeutic potential of RNA-based medicines for rare diseases.

Watch the full interview with Prof. Daniel Scherman:

Interview Questions:
Q1. Prof. Scherman,you have long been engaged in rare diseases, gene therapy, and gene delivery systems, while also actively contributing to translational research and international academic exchange. what do you see as the most critical bottlenecks and most promising directions in translating rare disease research from basic science into clinical applications during the Conference?
Q2. In your presentation on RNA-Based Therapeutics, considering ongoing challenges such as delivery efficiency, tissue specificity, and long-term safety, which technological approaches do you believe hold the greatest potential for breakthrough? How might these advances reshape future therapeutic strategies for rare diseases?

About the Interviewee: