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The frameshift variant NM_003986.3:c.675delA (p.Gly227Valfs*6) (family 1) leads to a premature stop codon and is predicted to code for a truncated protein of 231 amino acids (40% of the C-terminus sequence removed)

Changes in serum cytokine concentrations and the relationship between cytokine gene variants and the clinical course of the disease have been described in WD patients, as well as in animal models of this disease

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Among 38 identified hits, we found a subset of chloroacetamides with broad senolytic activity

Similar strategies may also be used to deplete IgA, as explored with the pre-clinical candidate BHV-1400 [434], which leverages ASGPR biology in a manner similar to BHV-1300 and could represent an alternative to the likely mechanism-of-action of ARGX-121, and to deplete IgE, as shown with lysosomal targeting chimeras coupling pH-sensitive target binding and ASGPR-mediated endolysosomal delivery to FcRn-mediated cellular transport of the potential therapeutic molecule [435]