Fig. 5: Ataluren reduced the levels of phospho-mTOR (S2248) in leukocytes from patients with SDS. | Nature Communications

Fig. 5: Ataluren reduced the levels of phospho-mTOR (S2248) in leukocytes from patients with SDS.

From: Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series

Fig. 5: Ataluren reduced the levels of phospho-mTOR (S2248) in leukocytes from patients with SDS.

Peripheral blood samples were collected from healthy donors (HD, white bars) and SDS patients (gray bars) before (0, dark gray), and after 3 and 6 months of therapy. Phospho-mTOR S2448 levels were analyzed by Phospho-Flow analysis in whole blood. Immunophenotypic markers were used to isolate different leukocyte populations. Data represent phospho-mTOR level in white blood cells (WBC) (a), lymphocytes (b), monocytes (c), and polymorphonuclear granulocytes (PMN) (d) of healthy donors (HD, n = 6) and SDS patients (n = 3, mean age 16.3, range 13–20) UPN26 (red lines, solid dot), UPN58 (blue lines, hollow dot), and UPN74 (green lines, inverted solid triangle). Data are mean ± SEM.

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