Enabling Novel CRISPR-Cas9 Delivery: A Case Study Supporting Innovative Genomics Institute

Dr. Jennifer Doudna and her team at the Innovative Genomics Institute (IGI) have partnered with Aldevron to advance their research on using CRISPR-based genome editing to treat Huntington's disease (HD) and amyotrophic lateral sclerosis (ALS). Their goal is to develop a non-viral delivery system that can effectively edit neurons with minimal immune responses.

To achieve this, they required a custom, highly pure, ultra-low endotoxin nuclease. Aldevron stepped in and provided their expertise in Cas9 expression and purification to meet this need. The resulting transient Cas9 RNPs demonstrated comparable editing of neurons and reduced immune responses compared to using AAV serotype 9 for delivery.

This exciting research suggests that injection-based delivery of CRISPR genome editing RNPs into the central nervous system could be a promising alternative to virus-mediated genome editing. Review how by partnering with Aldevron, Dr. Doudna and her team have made significant progress in their quest to develop new treatments for HD and ALS.