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CAR-T, Chimeric Antigen Receptor T-cell

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Since this is a genetic condition , I recommend screening siblings of affected patients, even if they have no symptoms

References 1.Wolfe F., Smythe H

Results in the study demonstrate that IGF-1 induces the opposite effects since the IGF-1 deficit reduces the expression of glucose-6-phosphatase and phosphoenolpyruvate carboxylase-1

The advantage of this type of compounds, apart from the reduction in side effects, is their efficient metabolism to non-toxic products and a high affinity for specific molecular targets, which can potentially be associated with enhanced treatment efficacy, enabling the use of a relatively low dose to produce identical biological effects [3]