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For a more detailed background, you can refer to upcoming resources on what peptide therapy is (article pending publication)
sgRNA constructs and gene expressing codon optimized (OPT) plasmid constructs sgRNA control and constructs against CBS and L2HGDH were designed targeting GTATTACTGATATTGGTGGG (sgControl) (#230080, Addgene), GGAGCAGACAACCTACGAGG (sg CBS -1) (#230081, Addgene), TGGCAGTGCTGGCAGCACGG (sg CBS -2) (#230082, Addgene), GATATAGTCATCGTTGGTGG (sg L2HGDH -1) (#230083, Addgene), and CACCAGACTGGACATAACAG (sg L2HGDH -2) (#230084, Addgene) and constructed in lentiCRISPR v2-Blast (#83480, Addgene) for Blasticidin selection at a final concentration of 5 g/ml
It is structural density improvement
First, optimal dosing in humans remains undefined, with current practice based primarily on extrapolation from animal studies and anecdotal clinical experience rather than formal dose-finding studies
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