AVROBIO, Inc.
COMPOSITIONS AND METHODS FOR TREATING NEUROCOGNITIVE DISORDERS

Abstract:

Described herein are compositions and methods for treating a patient having or at risk of developing a neurocognitive disorder, such as Alzheimer's disease, Parkinson's disease, and/or a frontotemporal lobar dementia. Using the compositions and methods of the disclosure, a patient, such as an adult human patient, may be provided one or more agents that elevate the expression and/or activity levels of a protein or series of proteins whose deficiency is associated with the corresponding disease. Exemplary agents that may be used in conjunction with the compositions and methods of the disclosure for this purpose include cells, such as cells, that contain nucleic acids encoding the protein or proteins of interest, as well as vectors, such as viral vectors, encoding the protein or proteins of interest. Additional examples of such agents include the protein or proteins themselves, as well as interfering RNA molecules that stimulate their endogenous expression.