A toolkit for the genome

 

Horizon, a translational genomics company that provides products and services that support personalized medicine, will gain access to intellectual property related to a proprietary artificial serotype of adeno-associated virus (AAV), which is commonly used for creating viral vectors and human disease models. The use of this AAV serotype will allow Horizon to target an increased range of cell types for homologous recombination.

 

“There are various serotypes you can use for purposes of gene editing, and we’ve tested quite a few of them, but this serotype from Stanford seems to be the one that has the highest applicability and allows for a broader testing mode for use in most cell lines,” Eric Rhodes, chief technology officer of Horizon Discovery Group, tells DDNews.

 

Stanford’s artificial serotype, known as AAV-DJ, was created by Prof. Mark Kay, director of the university’s human gene therapy program. The serotype has been shown to infect more than 80 percent of mammalian cells, which is significantly more than any naturally occurring AAV serotype.

 

The intellectual property from Stanford will supplement Horizon’s existing exclusive license for use of recombinant adeno-associated virus (rAAV) from the University of Washington. Horizon is the sole source of this mechanism for gene editing, which it claims is over 1,000 times more efficient at gene targeting in human somatic cell-types than plasmid-based methods. The company uses rAAV to initiate homologous recombination in cells, allowing any sequence alterations contained within the homologous DNA to be recombined with high precision into the target gene. Horizon anticipates that the high applicability of AAV-DJ will enable this process to be used more effectively in a broader range of cells.

 

“Horizon is committed to ensuring our customers are supported by the widest range of gene-editing tools secured by appropriate licenses, enabling us to offer the most complete suite of gene-editing products and services to meet their needs,” says Rhodes.

 

The new serotype will become part of the GENASSIST range of gene-editing kits that Horizon launched earlier this year. The GENASSIST kits provide off-the-shelf reagents for using CRISPR editing technology and a custom-tailored combination of these reagents to allow customers to generate their own CRISPR-ready cell lines. CRISPR is an RNA-guided gene-editing platform that makes use of a bacterially derived protein and a synthetic guide RNA to introduce a double-strand break at a specific location within the genome.

 

“When you want to knock in a specific mutation or element into a gene, you have to provide DNA that you want to insert in some form,” says Rhodes. “There are various options, but we’ve found that using AAV in combination with CRISPR creates a particularly efficient donor.”

 

Horizon provides its gene-editing kits for a wide range of customers, including biotech and pharmaceutical companies as well as academic institutions. “Most of these kits are custom-made to enable the customer to go after a particular gene that they are targeting,” says Rhodes. “We can put AAV-DJ into a kit with CRISPR agents and other tools, so customers can access all the components they need to do gene editing.”

 

This summer Horizon launched a program that allows academic researchers to access GENASSIST for free as part of a CRISPR knockout generation program. Horizon hopes that providing this free access will help promote the use of the CRISPR technology and expand the range of knockout cell lines available as disease models and compound screening tools. Researchers who participate in the program will be given free guide design and cloning into an all-in-one vector expressing CRISPR-associated protein 9.