ADENO-ASSOCIATED VIRAL VECTORS FOR TREATMENT OF MYOCILIN (MYOC) GLAUCOMA

摘要

FIELD: medicine. ;SUBSTANCE: group of inventions relates to the field of medicine and namely to ophthalmology and is intended for the treatment of an ocular disorder associated with the trabecular reticulum of the eye. A method for delivering nucleic acid into the trabecular reticulum of a mammalian eye includes introducing an AAV serotype 2 (AAV2) particle containing an rAAV vector into a mammalian eye. The rAAV vector contains nucleic acid and the AAV2 particle contains the AAV2 capsid protein containing the amino acid substitution R471A, the numbering is based on the VP1 of AAV2. Also provided is a method of treating an ocular disorder associated with the trabecular reticulum of the eye of a mammal. The method includes administering an AAV2 particle containing the rAAV vector to a mammalian eye. According to another embodiment, a kit is provided for delivering nucleic acid to the trabecular reticulum of a mammalian eye including an rAAV2 particle containing the rAAV vector. ;EFFECT: use of the group of inventions makes it possible to increase the effectiveness of the treatment of an eye disorder associated with the trabecular reticulum of the eye. ;18 cl, 16 dwg, 6 ex