Gene Therapy

Non-viral delivery vectors for gene therapy and genetic vaccination – moving beyond mRNA

Gene Therapy for Healthy Longevity Translational Research Programme

Dr Volker Patzel’s lab is developing and improving platform technologies with a focus on various kinds of RNA technologies and minimalistic non-viral dumbbell-shaped DNA delivery vectors.

Within the frame of our resources and appropriate arrangements, they can provide:

  1. Design and selection of functional RNA including antisense RNA (also ASOs), small interfering and small hairpin RNA (siRNA, shRNA), tertiary structure-stabilised hammerhead ribozymes, single-guide RNAs (sgRNA) for CRISPR editing, expression-improved mRNA and trans-splicing RNA for cell type-specific gene expression. The functions of all these types of RNAs can typically be improved by one or several orders of magnitude.
  2. Dumbbell-shaped DNA delivery vectors for coding or non-coding RNA expression in microgram scale. Dumbbells with sizes between 130 bp and 8000 bp have been manufactured and tested so far. Dumbbell production on a milligram scale may be available through our company AVECRIS.
  3. Within the frame of research collaborations, we are keen to explore targeting strategies for our nucleic acid-based mitochondrial delivery vectors.

 

For more information, email us: micvp@nus.edu.sg