Abstract: The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.

Abstract: Disclosed herein are methods of administering precision gene therapy, treating and/or preventing Alzheimer' disease progression, and reducing expression of APOE and APOE e4. Disclosed herein are isolated nucleic acid molecules, viral vectors, lentiviral vectors, pharmaceutical formulations, host cells, guide RNAs and plasmids for use in the disclosed methods.

Abstract: The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.

Abstract: The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.

Abstract: Disclosed herein are Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) 9-based epigenome modifier compositions for epigenomic modification of a SNCA gene and methods of use thereof.

Abstract: Disclosed are delivery platforms for use in gene editing that include a relatively short, highly efficient promoter that drives transcription of a nucleic acid sequence that encodes a gene-editing molecule, e.g., either a gRNA or a nuclease. In conjunction with this promoter, the vector includes one or more transcription factor binding elements (an Sp1 binding element and/or an NF-?B binding element) cloned into the vector upstream of a promoter that drives transcription of a gene-editing molecule. The vector can be a all-in-one CRISPR/Cas9 delivery platform and can incorporate one or more of the transcription factor binding elements upstream of a promoter for the gRNA component and/or of a promoter for the nuclease component.