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Role of iPSCs in Disease Modeling: Gaucher Disease and Related Disorders
Published in Deepak A. Lamba, Patient-Specific Stem Cells, 2017
Daniel K. Borger, Elma Aflaki, Ellen Sidransky
Treatment options for LSDs are generally limited and are, in some cases, restricted to palliative care and physical therapy (26). Enzyme replacement therapy (ERT), a treatment modality whereby the deficient enzyme is provided via intravenous infusions, is now available for several of the LSDs. While ERT is effective in treating the visceral and musculoskeletal manifestations of these diseases, generally, the infused enzymes are too large to cross the blood–brain barrier. As a result, ERT is ineffective at reversing brain involvement in neuronopathic LSDs. Even when ERT is an effective treatment option, it usually involves biweekly infusions for the remainder of the patients’ life, making it inconvenient and extremely expensive (more than US$ 200,000 per year). While, individually, the LSDs are very rare, collectively, they affect between 1 in 4000 and 1 in 8000 live births (27–29), meaning that the treatment of LSDs can still constitute a substantial burden on health care systems (30). These realities make the development of less expensive, neurologically active, and orally available small-molecule drugs an attractive goal for those researching LSDs.
Biocatalytic Nanoreactors for Medical Purposes
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2019
Oscar González-Davis, Chauhan Kanchan, Rafael Vazquez-Duhalt
A significant research effort is currently focused on the use of nanoparticles (NPs) for drug delivery. The design of nanocarriers (NCs) containing a specific drug that could be targeted to specific tissues, reducing the doses of drug needed by increasing the treatment efficiency is the primary goal of these smart drug delivery systems. Nevertheless, the NPs could also be used as NCs for enzymatic activity. The treatment of disorders originated from enzymatic activity deficiencies using therapeutic enzymes was first described 50 years ago (Duve, 1966). There are many diseases originated from the lack of one or more enzymatic activities, and because of this, in some cases, the administration of exogenous enzymes has been successfully used as enzyme replacement therapy (ERT).
Investigating the gas holdup in a gas-liquid cyclonic flotation column through the electrical resistance tomography
Published in International Journal of Coal Preparation and Utilization, 2022
Xiaokang Yan, Xiaoheng Li, Juan Li, Lijun Wang, Haijun Zhang, Yijun Cao
Further investigations reveal that ERT can not only provide real-time images of the void fraction on the internal cross-sections but also give quantitative information without disturbing the flow field. Vadlakonda & Mangadoddy (2017) applied ERT and explored the gas-liquid flow behavior in terms of the local and mean gas holdup in a 100 mm laboratory flotation column and evaluated the accuracy of ERT measurements by pressure transducers. Finally, they compared the measured gas holdup by ERT with the results obtained from the numerical simulation. More recently, Vadlakonda & Mangadoddy (2018) coupled ERT with pressure transducers and studied the hydrodynamic characteristics of three-phase flows in the column flotation. It was found that the measured mean gas holdup and solid holdup by ERT are in good agreement with the obtained data from pressure difference measurements. Vakamalla et al. (Vakamalla et al. 2017) successfully explored the effect of air superficial velocity and liquid flow rate on the mean gas holdup and radial distributions and studied the hydrodynamics of the hydrocyclone mineral separators through ERT and CFD simulations. The performed literature review indicates that ERT can be effectively applied in diverse applications. Meanwhile, it is a safe and non-invasive scheme with high time resolution. Considering the superior characteristics of the ERT method, it is an appropriate scheme to measure the gas holdup in FCSMC with large variations within a short period and accompanied by the rotating flow of bubbles.