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Allelic variants for HhRz Therapeutics | |
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5′ UT Target Site | |
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5′…CCUGAGUGGCUGAGCUC AGGCCUU | (5′ UT target site CUC) |
5′…CCUGAGUGGCUGAGCUG AGGCCUU | (aWT variant mRNA, CUG cannot be cleaved) |
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Coding V230 region | |
5′…CUC GUC UUC ACC GUC AAG GAG GCC…3′ | (Coding region GUC) |
L226 V227 F228 T229 V230 K231 E232 A233 | (Amino acid triplets) |
5′…CUC GUC UUC ACC GUG AAG GAG GCC…3′ | (WT variant mRNA, |
L226 V227 F228 T229 V230 K231 E232 A233 | GUG cannot be cleaved) |
Single letter amino acid codes are used. | |
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Coding F293 region | |
5′…AUC CCA GCG UUC UUU GCC AAG AGC…3′ | (Coding region GUU) |
I290 P291 A292 F293 F294 A295 K296 S297 | |
GUU cleavage site occurs within the F293 codon rather than cutting at the end of a codon. | |
5′…AUC CCA GCG UGC UUU GCC AAG AGC…3′ | (F293C variant mRNA) |
I290 P291 A292 C293 F294 A295 K296 S297 | |
It is unclear whether or not the F293C mutation is an allelic variant WT or has a protein phenotype. | |
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Allelic variant for RNAi therapeutics in F293 region | |
5′…AUC CCA GCG UUC UUU GCC AAG AGC…3′ | (Coding region RNAi site) |
I290 P291 A292 F293 F294 A295 K296 S297 | |
5′…AUA CCC GCA UUU UUC GCG AAA AGG…3′ | (WT variant mRNA) |
I290 P291 A292 F293 F294 A295 K296 S297 | |
aWT variant generated by codon degeneracy across the region of designed RNAi antisense annealing. | |
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