Name: Michael Przybilla, Ph.D.

Title: The PS Gene Editing System: A Platform Technology for Treating Lysosomal Disorders

Description: The PS Gene Editing System (PSG System) is an AAV-based gene editing platform technology developed as a single-dose treatment for monogenic disorders, in particular lysosomal diseases. The PSG System employs CRISPR-Cas9 to generate a targeted double-stranded DNA break in the first intron of the albumin locus in hepatocytes. By providing a second AAV containing a donor template encoding a functional copy of the lysosomal enzyme cDNA, these hepatocytes effectively become an enzyme-producing factory. In this presentation, data will be presented that demonstrates the efficacy of this system as a therapeutic for lysosomal disorders such as GM1-gangliosidosis.

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