Controlled release delivery systems play essential roles in gene therapy and gene editing applications. During the past few decades, great advances have been made in the delivery of nucleic acids to generate therapeutic proteins (e.g. pDNA, mRNA) and to eliminate disease causing proteins (e.g. siRNA, miRNA, ASOs). More recently, the development of gene editing technologies including CRISPR/Cas, TALENs, and zinc finger nucleases (ZFNs) has opened extraordinary opportunities to precisely edit the genome. Because of the ability to target disease causing mutations, these methods hold incredible promise for one-time cures of genetic diseases. Despite this potential, therapeutic success still largely depends on the development of viral and non-viral carriers to deliver and edits genes within targeted cells. Current efforts in the gene delivery and editing community are focused on creating a better fundamental understanding of the barriers to delivery, the design of improved carriers, and realizing opportunities for therapeutic intervention. This is particularly true for emerging therapeutics including mRNA and CRISPR/Cas, for which much remains unknown. There is also a present challenge to develop viral and non-viral carriers that can deliver and edit genes in specific organs and cells. Thus, a great future lies ahead for the field of gene delivery and editing, while equally great challenges also stand in the way. The Gene Delivery and Editing Focus Group will serve investigators from universities and research institutes with scientific interests on development of viral and non-viral carriers, examination of delivery barriers, fundamental and applied gene delivery and gene editing applications, industrial researchers from companies with R&D in the vast fields of protein therapies, gene therapies, gene silencing, and gene editing, and experts from regulatory bodies, each under the umbrella of CRS.
The members of the Focus Group are expected to be paid members of the Controlled Release Society, independent of their membership category.
- MRNA Vaccines and Therapeutics: From Promise to Reality - Orn Almarsson, PhD - March 2, 2020 2PM EST