GENE DELIVERY METHODS AND COMPOSITIONS

摘要

The invention provides methods and compositions that remove target genetic material from a subject by hydrodynamic delivery of an enzyme that degrades the target genetic material. The methods include delivering a solution include a nucleic acid, protein, or ribonucleoprotein to a blood vessel at a pressure sufficient to cause the material to enter cells proximal to the blood vessel. The enzyme may be Cas9 and may be provided with a guide RNA. The target genetic material may be viral genome with the guide RNA complementary to a portion of the viral genome.

主权项

1. A system for delivering a nuclease, the system comprising:
a solution comprising a targetable nuclease, wherein the targetable nuclease comprises an enzyme that cuts target genetic material in a sequence-specific manner; a delivery catheter dimensioned for insertion into a blood vessel of a subject; and a delivery mechanism operable to deliver the solution through the catheter at a pressure sufficient to cause the targetable nuclease to enter cells proximal to the blood vessel.