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Genome Editing and Gene Therapies: Complex and Expensive Drugs
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2020
These diseases are inherited hemoglobin disorders resulting from reduced functional hemoglobin. Both are monogenic diseases with a large spectrum of phenotypes due to genetic modifiers in SCD (Driss et al., 2009) or a large number of mutations in the I3-globin gene or associated regulatory elements (RiveIla, 2015). According to the WHO (Modell and Darlison, 2008) it is estimated that “1.1% of couples worldwide are at risk for having children with a hemoglobin disorder;’ a number that is rising steadily. A management of SCD includes hydroxyurea that reduce the frequency of painful episodes and pain control such as opioid administration or NSAIDs in milder cases. Hydroxyurea, developed as an anticancer drug, in addition raises the level of fetal hemoglobin (HbF; Œ2y2) and that of total hemoglobin by a mechanism not fully understood so far, and reduces the white blood cell count (Agrawal et al., 2014). The beneficial effects of HbF in sickle cell anemia have been reviewed by Akinsheye et al. (2011). Recent findings concerning the mechanism of switching from fetal to adult hemoglobin have been reported by Wang and Thein (2018).
Multiresolution functional characterization and correction of biofouling for improved biosensing efficacy
Published in IISE Transactions, 2023
Weizhi Lin, Cesar Ruiz, Matan Aroosh, Hadar Ben-Yoav, Qiang Huang
In this section, we apply the proposed biofouling correction methodology to an Hydroxyurea (HU) detection experiment using three consecutive measurements. Hydroxyurea (HU) is an oral medication for treating sickle cell anemia, and its blood concentration level is related to treatment efficacy and compliance. In practice, the HU level is determined by High-Performance Liquid Chromatography (HPLC) (Bachir et al., 2007). A large amount of fluid is required for HPLC to separate, identify, and quantify each component in the mixture. The detection is expensive and time-consuming for patients, which might delay early treatment adjustments. Therefore, on-the-spot inspection is vital for adequate treatment planning (Cazelles et al., 2021). However, biofouling-induced voltammogram changes reduce prediction accuracy even for repeated measurements. In this section, we correct both the second and third measured voltammograms and evaluate the correction efficacy by signal-wise MARE and prediction accuracy of a scalar-on-functional model.
Evaluation of the developmental toxicity of solvents, metals, drugs, and industrial chemicals using a freshwater snail (Biomphalaria glabrata) assay
Published in Journal of Toxicology and Environmental Health, Part A, 2022
Rosângela Ribeiro de-Carvalho, Maria Regina Gomes-Carneiro, Barbara Rodrigues Geraldino, Gabrielle da Silveira Lopes, Francisco José Roma Paumgartten
It is of noteworthy that the snail assay confirmed the embryotoxicity of colchicine and hydroxyurea (LOECs 0.2 and 5 μM, respectively) noted in lab rodents (Chahoud and Paumgartten 2009; Sieber et al. 1978). Colchicine crosses the placenta and high doses of this alkaloid were reported to be embryotoxic and teratogenic to rodents (Michael et al. 2003; Sieber et al. 1978), although its therapeutic use in pregnancy has not been associated with miscarriages and/or birth defects (Indraratna et al. 2018; Michael et al. 2003). Similarly, hydroxyurea is overtly teratogenic in rodent models (Chahoud and Paumgartten 2009). Although not recommended for use in pregnancy, hydroxyurea has not been associated with an excess of risk of birth defects when therapeutic doses were administered to pregnant women (Ballas et al. 2009).
Potential application of XC3 (X = B, N) nanosheets in drug delivery of hydroxyurea anticancer drug: a comparative DFT study
Published in Molecular Physics, 2022
Rezvan Rahimi, Mohammad Solimannejad, Zeynab Ehsanfar
Hydroxycarbamide (HC), or the anticancer drug hydroxyurea, has been used as an anti-neoplastic drug in myeloproliferative disorders, mainly polycythemia Vera and essential thrombocythemia. The hydroxyurea anticancer drug restrained the growth of cancer cells in the head and neck, and it cures many neoplastic diseases, including chronic myelogenous leukemia [16–18]. HC anticancer drug used in the treatment of sickle cell anemia [19]. Some of the side effects of this drug are drowsiness, nausea, diarrhea and vomiting, constipation, mucositis, anorexia, stomatitis, bone marrow toxicity, skin changes, abnormal liver enzymes, creatinine, and blood urea nitrogen [20]. These side effects may happen when interacting with the wrong purpose or in the incorrect tissue. One compelling method to minimise drugs’ side effects and toxicity is to send prescriptions to the target regions. Drug delivery technologies make it possible to increase the efficiency of drug uptake and distribution.