Abstract: This disclosure provides CRISPR/Cas9 based fusion molecules and guide RNAs for use in in vivo targeted reduction or elimination of VEGFA gene products. This disclosure also relates to formulations, methods of production and methods of use thereof.

Abstract: The invention provides for a composition, which in particular comprising a fusion molecule comprising a least one DNA binding protein and at least one modulator of gene expression, wherein the fusion molecule is targeted to a genomic region near a VEGF gene and/or within a VEGF regulatory element and the at least one modulator of gene expression provides a modification of at least one nucleotide near the VEGF gene and/or within a VEGF regulatory element. The invention also provides methods of reduction or elimination of VEGF gene products in vivo and uses thereof.

Abstract: The present invention provides a model or tool for predicting the editing efficiency of sgRNA in a CRISPR/Cas gene editing system, in particular a CRISPR/dCas epigenetic editing system, a training and prediction method thereof, and a related computer system, computer storage medium, and application. In particular, one or more sgRNA and target gene related epigenetic features are added to an input of the model.

Abstract: This disclosure provides CRISPR/Cas9 based fusion molecules and guide RNAs for use in in vivo targeted reduction or elimination of VEGFA gene products. This disclosure also relates to formulations, methods of production and methods of use thereof.

Abstract: This disclosure provides CRISPR/Cas9 based fusion molecules and guide RNAs for use in in vivo targeted reduction or elimination of PCSK9 gene products. This disclosure also relates to formulations, methods of production and methods of use thereof.