EP 3625338 A1 20200325 - ENGINEERING OF A MINIMAL SACAS9 CRISPR/CAS SYSTEM FOR GENE EDITING AND TRANSCRIPTIONAL REGULATION OPTIMIZED BY ENHANCED GUIDE RNA
Title (en)
ENGINEERING OF A MINIMAL SACAS9 CRISPR/CAS SYSTEM FOR GENE EDITING AND TRANSCRIPTIONAL REGULATION OPTIMIZED BY ENHANCED GUIDE RNA
Title (de)
KONSTRUKTION EINES DURCH ERWEITERTE GUIDE-RNA OPTIMIERTEN MINIMALEN SACAS9-CRISPR/CAS-SYSTEMS ZUR GENEDITIERUNG UND TRANSKRIPTIONSREGULIERUNG
Title (fr)
INGÉNIERIE D'UN SYSTÈME CRISPR/CAS MINIMAL SACAS9 POUR L'ÉDITION DE GÈNES ET LA RÉGULATION TRANSCRIPTIONNELLE OPTIMISÉE PAR UN ARN GUIDE AMÉLIORÉ
Publication
Application
Priority
CN 2017085202 W 20170519
Abstract (en)
[origin: WO2018209712A1] Programmable and precise regulation of Cas9 functions by utilizing a set of compact Cas9 derivatives created by deleting conserved HNH and/or REC-C domains based on the structural information across variant class 2 CRISPR effectors is provided. A novel strategy for engineering the dimeric gRNA-guided nuclease by splitting the mini-dSaCas9 and fusing the FokI domain right after the split point to increase the on-target DNA cleavage efficiency and potentially reduce the off-target effect because of a closer proximity of dimeric FokI nuclease to the target sequence is also provided. By combining the optimized and compact gRNA expression cassette and the downsized SaCas9 derivatives, the entire CRISPR/Cas system with different effector domains for transactivation, DNA cleavage and base editing is loaded into a single AAV virus. Such an all-in-one AAV-CRISPR/Cas9 system will be particularly appealing in biomedical applications that require safe and efficient delivery in vivo.
IPC 8 full level
C12N 9/22 (2006.01); C12N 15/115 (2010.01)
CPC (source: EP)
C12N 9/22 (2013.01); C07K 2319/00 (2013.01); C12N 2310/20 (2017.04)
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated extension state (EPC)
BA ME
DOCDB simple family (publication)
WO 2018209712 A1 20181122; CN 110662835 A 20200107; CN 110662835 B 20230428; EP 3625338 A1 20200325; EP 3625338 A4 20210120
DOCDB simple family (application)
CN 2017085202 W 20170519; CN 201780091204 A 20170519; EP 17910146 A 20170519