Home » SiR-A novel vector for CRISPR delivery into the CNS

SiR-A novel vector for CRISPR delivery into the CNS

Modality

Advanced therapies

Therapeutic area

Neuroscience

Advantages

The vector allows for a highly site specific level of infection at a very low concentration. Moreover, retrograde movement of Rabies virus means the vector could be delivered at the spinal cord or peripheral motor neurones, before tracing to a specific cell type deeper in the CNS.

Background info

A neurotropic, attenuated Rabies virus vector that can be used to deliver a payload to the CNS before self-inactivating, thereby allowing for gene editing.

Indication

Central nervous system disorders

Intellectual property

Granted patents and pending patent applications that entered their respective national stage from WO2018203049