Genome editing, a mechanism to rewrite DNA sequences, has the potential to create a new generation of precise genetic therapies that can ameliorate or cure genetic disorders. For genome editing to be therapeutic, it must not only produce the desired genetic modifications, but also efficiently target the appropriate cells in the appropriate organs. Ex vivo and in vivo delivery approaches are the main platforms used to achieve modification of target tissues. While ex vivo modification of hematopoietic stem cells is being primarily developed to target genetic diseases of the blood such as hemoglobinopathies and immune deficiencies, it also has great potential to treat non-hematological multi-systemic diseases. In this webinar we will discuss how the hematopoietic system can be engineered using genome editing to produce, secrete, and deliver therapeutic proteins to multiple organs, including the central nervous system. Specifically, we discuss recent data, and provide specific examples, regarding how such an approach can become a therapeutic platform for a large group of genetic diseases caused by deficiencies in lysosomal enzymes. We also review a CRISPR/Cas9-based, good-manufacturing-practice-compatible protocol for modification of hematopoietic stem cells ex vivo. 

 

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