Aldevron, Jennifer Doudna, and Innovative Genomics Institute Collaborate on In Vivo CRISPR Therapies for Neurologic Diseases. Resulting paper published in Molecular Therapy.
FARGO, N.D. (August 29, 2023)— Aldevron, a leading global manufacturer of DNA, RNA and proteins used in cell and gene therapies and vaccine development, contributed to a publication in collaboration with 2020 Nobel Laureate Jennifer Doudna and the Innovative Genomics Institute (IGI) at the University of California, Berkeley to advance research on in vivo, CRISPR-based genome editing strategies to address genetic diseases of the central nervous system such as Huntington’s disease and ALS. Aldevron supplied custom CRISPR protein with ultra-low endotoxin, enabling direct delivery to the brain in mice. This partnership resulted in a paper, titled “Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs,” published in the journal Molecular Therapy, the official journal of the American Society of Gene and Cell Therapy.