The process that turns coded information in DNA into proteins requires two steps: transcription, in which DNA is used to generate a complementary RNA strand by RNA polymerase, and translation, in which RNA is used to produce a protein by ribosomes. Disrupting either step can slow or prevent the expression of a mutant gene. Ataxin 1 is involved in a number of signaling pathways and its expression is controlled by signaling pathways. The MAPK/ERK pathway has been shown to activate ataxin 1 expression, and MSK1 also phosphorylates ataxin 1, controlling its localization and degradation. Inhibitors of key proteins in this pathway may be used in combination therapy to potentially decrease expression and lower steady state concentrations of ataxin 1.One technique for disrupting translation, antisense oligonucleotide therapy, which uses single strands of RNA complementary to the target to prevent the target from binding to a ribosome and trigger the degradation of the target, has already begun clinical trials in other neurodegenerative disorders with many different delivery mechanisms. A similar technique is RNA interference or RNAi. Instead of complementary antisense strands of RNA, RNAi uses very small double stranded segments of RNA called small interfering RNA which triggers degradation of the target before it can be translated. Studies using RNAi agents delivered by adeno associated viruses (AAV) has been shown to halt progression of disease and lead to some recovery of function with treatment applied to only the deep cerebellar nuclei in mice and rhesus macaques.
Lactitol is a sugar alcohol used as a replacement bulk sweetener for low calorie foods with 30–40% of the sweetness of sucrose. It is also used medically as a laxative. Lactitol is produced by two manufacturers, Danisco and Purac Biochem. Applications
Lactitol is used in a variety of low food energy or low fat foods. High stability makes it popular for baking. It is used in sugar-free candies, cookies (biscuits), chocolate, and ice cream, with a sweetness of 30–40% that of sucrose. Lactitol also promotes colon health as a prebiotic. Because of poor absorption, lactitol only has 2–2.5 kilocalories (8.4–10.5 kilojoules) per gram, compared to 4 kilocalories (17 kJ) per gram for typical saccharides. Hence, lactitol is about 60% as caloric as typical saccharides. Medical
Lactitol is listed as an excipient in some prescription drugs.Lactitol is a laxative and is used to prevent or treat constipation, e.g., under the trade name Importal.In February 2020, Lactitol was approved for use in the United States as an osmotic laxative for the treatment of chronic idiopathic constipation (CIC) in adults.Lactitol in combination with Ispaghula husk is an approved combination for idiopathic constipation as a laxative and is used to prevent or treat constipation. Safety and health
Lactitol, erythritol, sorbitol, xylitol, mannitol, and maltitol are all sugar alcohols. The U.S. Food and Drug Administration (FDA) classifies sugar alcohols as "generally recognized as safe" (GRAS). They are approved as food additives, and are recognized as not contributing to tooth decay or causing increases in blood glucose.