The Leap of Inulin Fructans from Food Industry to Medical Application.

As a dietary supplement, the efficacy of prebiotics has become a hot issue in recent years. Inulin is one of internationally recognized prebiotics and belongs to a group of non-digestible and fermentable carbohydrates. Currently, the food industry is increasingly using prebiotic inulin as a health-promoting substrate, not just as food supplement. In addition, inulin has also shown great promise in the treatment of various diseases. This article reviews the application of inulin in the food industry and summarizes physiological function of inulin. Through the review and prospect of the research on obesity, diabetes and mental illness, it provides the theoretical basis for the joint development of inulin in food industry and medical application.

Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology.

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is caused by reduced levels of functional survival motor neuron (SMN) protein. To identify therapeutic agents for SMA, we established a versatile SMN2-GFP reporter line by targeting the human SMN2 gene. We then screened a compound library and identified Z-FA-FMK as a potent candidate. Z-FA-FMK, a cysteine protease inhibitor, increased functional SMN through inhibiting the protease-mediated degradation of both full-length and exon 7-deleted forms of SMN. Further studies reveal that CAPN1, CAPN7, CTSB, and CTSL mediate the degradation of SMN proteins, providing novel targets for SMA. Notably, Z-FA-FMK mitigated mitochondriopathy and neuropathy in SMA patient-derived motor neurons and showed protective effects in SMA animal model after intracerebroventricular injection. E64d, another cysteine protease inhibitor which can pass through the blood-brain barrier, showed even more potent therapeutic effects after subcutaneous delivery to SMA mice. Taken together, we have successfully established a human SMN2 reporter for future drug discovery and identified the potential therapeutic value of cysteine protease inhibitors in treating SMA via stabilizing SMN proteins.