Phenylketonuria (PKU) is an inborn error in the metabolism of phenylalanine (phe) and tyrosine (Tyr). It is an autosomal recessive disease caused by a deficiency of the liver enzyme phenylalanine hydroxylase (PAH). Patients with PKU are unable to metabolize the amino acid phe, resulting in its accumulation throughout the body. If left untreated, elevated Phe levels can lead to severe neurological complications and disability. The goal of PKU treatment is to reduce blood phe to a level that allows normal brain development. Metabolic disorders, such as PKU require lifelong treatment. Therefore, the development of low-cost and easy-to-administer therapeutic strategies is essential to increase individual compliance with treatment to increase their effectiveness. Next-generation probiotics are living biotherapeutics that have been genetically engineered to target specific diseases. It is now possible to engineer the composition of therapeutic molecules or enzymes expressing probiotics with multiple beneficial properties, potentially increasing their potential beneficial effects against a wider range of diseases. In the case of individuals with PKU, a new option for their treatment is the use of genetically modified probiotics, which is a scientific advancement in genetic engineering, for the production and release of metabolic enzymes effective in reducing blood concentrations of Phe. PKU is another example of a metabolic disorder that may benefit from the use of probiotics to deliver the PAL enzyme to the gut.
Fig.1 An overview of how probiotics can improve symptoms of PKU.1
Research Articles | Available Services |
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Genetically engineered probiotic for the treatment of PKU; assessment of a novel treatment in vitro and in the PAHenu2 mouse model of PKU.2 |
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Adaptive Strategies of the Candidate Probiotic E. coli Nissle in the Mammalian Gut.3 |
Probiotic strains are attractive for the development of biotherapeutics. For developing successful therapies for the treatment of PKU, it is important to consider characteristics such as genetic stability. More studies are needed to validate the use of these next-generation probiotics in humans, assess the effect of probiotic therapy on gut microbiota composition, and ensure their safety because they are genetically modified organisms. E. coli Nissle is an important strain that has been studied for the development of engineering therapies for PKU because it presents an excellent safety profile and genetic tractability. Creative Biolabs has a strong technical background in engineering strain construction and safety assessment. Please feel free to contact us to discuss your project, and we are happy to provide you with professional technical insights.
References
For Research Use Only. Not intended for use in food manufacturing or medical procedures (diagnostics or therapeutics). Do Not Use in Humans.
For Research Use Only. Not intended for use in food manufacturing or medical procedures (diagnostics or therapeutics). Do Not Use in Humans.
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