Materials and methods for treatment of glycogen storage disease type 1A
AssigneeCrispr Therapeutics AG
InventorsChad Albert Cowan, Roman Lvovitch Bogorad, Ante Sven Lundberg, Kirsten Leah Beaudry
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Abstract
The present application provides materials and methods for treating a patient with Glycogen Storage Disease type 1a (GSD1a) both ex vivo and in vivo. In addition, the present application provides materials and methods for modulating the expression, function, and/or activity of the glucose-6-phosphatase, catalytic (G6PC) and/or the glucose-6-phosphatase (G6Pase) protein in a cell by genome editing.
Claims6
One or more guide ribonucleic acids (gRNAs) for editing exon 1, exon 2 or exon 5 of a glucose-6-phosphatase, catalytic (G6PC) gene in a cell, wherein the one or more gRNAs comprise a spacer sequence that is 10 to 30 nucleotides in length, wherein the spacer sequence is an RNA sequence corresponding to the full-length nucleic acid sequence selected from the group consisting of SEQ ID NOs:
- 57,319, 57,306, 52,935, 57,305, 52,938, 52,940, 57,296, 52,951, 52,918, 57,320, 52,919, 52,923, 57,308, 57,295, 56,893, 53,372, 53,374, 56,890, 56,883, 56,881, 53,380, 56,879, 56,878, 53,382, 56,876, 56,865, 56,863, 56,909, 56,898, 53,370, 56,860, 55,845, 55,842, 54,541, 55,820, 54,560, 53,360, 53,362, 59,984, 53,358, 55,838, 52,962, 53,369, 57,300, 52,910, 57,321, 57,312, 52,939, 57,299, 52,944, 52,957, 57,274, 53,364, 53,373, 53,375, 56,882, 56,873, 53,392, 53,403, 53,363, 54,506, 55,875, 55,874, 55,873, 55,872, 54,511, 55,861, 55,837, 54,542, 54,544, 54,558, 64,798, 64,799, 64,801, 56,893, 64,808, 64,809, 64,810, 64,811, 64,812, 64,813, 53,372, and 64,816, wherein the one or more gRNAs has an editing efficiency of at least 10%.
Dependencies are inferred from the claim text.