Ex-Vivo and In-Vivo Genome Engineering

Improvements in sequencing technologies have increased our ability to diagnose genetic diseases, while effective interventions lag behind. The development of tools to edit DNA and the increased understanding of DNA repair mechanisms have enabled new therapeutic modalities for the treatment of genetic diseases. Site-specific genome editing can be used to correct disease-causative mutations, insert a functional copy of a defective gene, and ultimately modulate metabolic pathways by disrupting or enhancing the expression of key biochemical processes.

Several therapeutic strategies based on genome editing are currently tested in human clinical trials for the treatment of monogenic diseases. Ex-vivo genome editing of hematopoietic stem cells (HSC) is being tested for beta-Thalassemia and Sickle cell disease while in-vivo genome editing of retinal photoreceptor cells is being applied to Leber Congenital Amaurosis type 10 (LCA10). For metabolic diseases or inborn errors of metabolism, despite many promising proof-of-concept studies in animal models, only in-vivo hepatic genome editing has been tested clinically to convert the growing liver in a factory organ for enzymes deficient in lysosomal storage diseases (LSDs).

Metabolic diseases, also known as inborn errors of metabolism, are a large group of genetic diseases that represent a large unmet medical need. For many of these diseases, the pathophysiology is potentially trackable using genome editing. However, these diseases pose a unique set of challenges. Many are multi-systemic in nature and therefore require the targeting of multiple cell-types/organs. Some are rapidly progressive which augments the need for approaches that can provide rapid metabolic compensation. Finally, most individuals affected are children which poses additional operational and ethical complications for clinical testing. Like other monogenetic diseases the road to the clinic is still challenged by the low efficiency of in-vivo therapeutic editing in target cells and the limited availability of cells with regenerative potential for all affected organs that can be targeted ex-vivo.

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