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Treating the wrong target tends to give disappointing results
37 In one study of 13 kindreds with hereditary non-spherocytic hemolytic anemia and either single or no identified mutations in PKLR coding regions, whole genome sequencing identified five kindreds with unique PKLR deep intronic mutations predicted to perturb normal mRNA processing
BMC Psychiatry 19(1):305
6:26585 10.1038/srep26585 [DOI] [PMC free article] [PubMed] [Google Scholar] Lushchak V.I
