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Dendrimer Conjugates as Novel DNA and siRNA Carriers
Published in Raj K. Keservani, Anil K. Sharma, Rajesh K. Kesharwani, Drug Delivery Approaches and Nanosystems, 2017
Yuya Hayashi, Keiichi Motoyama, Taishi Higashi, Hirofumi Jono, Yukio Ando, Hidetoshi Arima
To clarify the potential use of Lac-α-CDEs (G3) as novel hepatocytespecific siRNA carriers, Hayashi et al. evaluated the RNAi effect of siRNA complexes with Lac-α-CDEs (G3) both in vitro and in vivo (Hayashi et al., 2012). Transthyretin (TTR)-related familial amyloid polyneuropathy (FAP), which is induced by amyloidogenic TTR (ATTR), is an autosomal dominant form of fatal hereditary amyloidosis characterized by systemic accumulation of amyloid fibrils in peripheral nerves and other organs (Ando et al., 2005). So, we targeted TTR gene expression, which mainly produced in hepatocytes. Lac-α-CDE (G3, DSL1) complex with TTR siRNA (siTTR) had the potent RNAi effect against TTR gene and protein expression through adequate physicochemical properties, ASGPR1-mediated cellular uptake, efficient endosomal escape and the delivery of the siRNA complex to cytoplasm, but not nucleus, with no cytotoxicity. The Lac-α-CDE (G3, DSL1) complex with siTTR had the potential to exert the in vivo RNAi effect after intravenous administration in the liver of mice. The blood chemistry values of Lac-α-CDE (G3, DSL1) system were almost equivalent to those of control system. Thus, these results suggest that Lac-α-CDE (G3, DSL1) has the potential for novel hepatocyte-specific siRNA carriers in vitro and in vivo, and has a possibility as a therapeutic tool for FAP. As described above, Lac-α-CDEs have the potential for a novel hepatocyte-specific DNA and siRNA carrier in vitro and in vivo (Hayashi et al., 2012). However, DNA and siRNA transfer activity of Lac-α-CDEs decreased in the presence of high concentration of serum in vitro. In addition, hepatocytes-specific transfer activity of Lac-α-CDE was not enough satisfactory in vivo, although it was superior to that of jetPEITM-Hepatocyte (Arima et al., 2010).
Skin temperature of the foot: comparing transthyretin Familial Amyloid Polyneuropathy and Diabetic Foot patients
Published in Computer Methods in Biomechanics and Biomedical Engineering: Imaging & Visualization, 2019
Adérito Seixas, Maria do Carmo Vilas-Boas, Rui Carvalho, Teresa Coelho, Kurt Ammer, João Paulo Vilas-Boas, Joaquim Mendes, João Paulo Silva Cunha, Ricardo Vardasca
Peripheral neuropathy is a condition affecting nerves at different levels. It may cause motor, sensory and autonomic impairments. In the foot, sensory neuropathy implies a loss of feeling, motor neuropathy may lead to foot deformities and inability to coordinate movements and autonomic neuropathy impairs sweat and sebaceous glands. All these factors increase the vulnerability of the foot to the development of infection and ulcer formation (Frykberg et al. 2006; Noor et al. 2015; Adams et al. 2016). Peripheral neuropathy is present in many disabling and life-threatening conditions such as Diabetes and Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP).
The roadmap towards cure of chronic hepatitis B virus infection
Published in Journal of the Royal Society of New Zealand, 2022
Translation inhibitors can silence production of disease-associated proteins and enzymes by intervening at the transcript level (mRNA) (Figure 3). Target diseases include both inherited conditions associated with unwanted gene products including hypercholesteraemia, familial amyloid polyneuropathy, PNH, acute intermittent porphyria and alpha-1-antitrypsin deficiency and acquired diseases such as chronic bacterial or viral infections.