Metagenomi products

Large gene integrations; Direct­ed DNA inte­gra­tion has large­ly been con­sid­ered the ulti­mate goal of cor­rec­tive genome edit­ing. This tech­nol­o­gy has the poten­tial to address a large col­lec­tion of com­plex genet­ic dis­eases caused by any loss of func­tion muta­tions, such as cys­tic fibro­sis, where inser­tions of greater than 10,000 bp would be required to address all muta­tions. Our CASTs are being devel­oped to per­form these com­plex gene integrations.

Multiple nucleases to edit any target in the human genome . Our tool­box con­tains thou­sands of CRISPR nucle­as­es allow­ing us to select the ide­al sys­tem for tar­get­ing any giv­en gene in a site-spe­­cif­ic man­ner, poten­tial­ly over­coming a major lim­i­ta­tion of first-gen­er­a­­tion CRISPR sys­tems. In order to mod­i­fy the genome, nucle­as­es are used to cre­ate tar­get­ed genom­ic breaks, trig­gering DNA repair path­ways. This allows us to inte­grate a gene at a tar­get site (knock-in), or deac­ti­vate a gene (knock-down).