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The use of hematopoietic cells in vitro is not limited to the research of immunological factors, patient-derived lymphocytes have also been employed in functional studies, identifying new pathogenic variants, and scanning for susceptibility of the variants to chaperone treatment in combination with in silico predictions of pathogenicity [49]

doi: 10.1016/j.ymthe.2024.05.037 49 ZhaoG

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