News
1.The first poster will be shown on Monday, May 16, 2022, describing the use of a glutaryl-CoA dehydrogenase (GCDH) deficient mouse model to evaluate the therapeutic effects of AAV-delivered GCDH gene replacement therapy in treating glutaric aciduria type I (GA-1). The rAAV9-hGCDH was intracerebroventricularly injected into the GCDH-/- neonates and GCDH protein expression was observed in the brain of the rAAV treated mice. After high protein diet (HPD) challenge, the rAAV treated GCDH-/- mice showed significantly increased survival rates.
2.The second poster will be presented on May 18th, 2022, describing an engineering method termed Improving Editing Activity by Synergistic Engineering (MIDAS), to significantly improve the mammalian genome editing activities of the CRISPR/Cas nucleases. MIDAS-engineered variants of Cas12i, Cas12b and CasX showed significantly increased editing efficiencies in human cells, up to 137-, 11-, and 465-fold, respectively. Notably, a Cas12i variant, termed Cas12iMax, exhibited robust activity with a very broad PAM range and significantly higher editing efficiency than the currently widely used Cas nucleases, e.g., AsCas12a, BhCas12b v4, SpCas9, SaCas9 and SaCas9-KKH.
