AAV5-Delivered Life Edit® CRISPR System Results In Broad CNS Biodistribution And Allele Selective Editing And Reduction Of mHTT Protein In Critical HD Brain Regions

By Logan Y. Brown, Ph.D.; Alexandra Crawley, Ph.D.; Ritika Jaini, Ph.D.; Hanqian Mao, Ph.D.; Jamie Moy, Ph.D.; and Kathryn Woodburn, Ph.D.

Life Edit’s genome editing platform provides an extensive and diverse collection of cutting-edge RNA-guided nucleases, base editors, and reverse transcriptase editors, offering highly flexible and precise editing strategies that unlock unprecedented access to a wide range of genomic loci.

These tools, known as Life Edit Genes (LEGs), are specifically designed with unique protospacer adjacent motif (PAM) recognition sequences, which significantly broaden the range of targetable sites across the genome. This flexibility allows for the targeting of diverse genomic loci, including many genes linked to genetic disorders, making it possible to address a wide variety of diseases through precision editing.

By leveraging these powerful technologies, Life Edit offers a platform that can be tailored to meet the needs of a broad array of therapeutic applications, from correcting specific mutations to introducing complex genetic alterations, enabling the next generation of genomic medicine.