scholarly journals Chelation therapy in liver diseases of childhood: Current status and response

2021 ◽  
Vol 13 (11) ◽  
pp. 1552-1567
Author(s):  
Jayendra Seetharaman ◽  
Moinak Sen Sarma
Cancers ◽  
2019 ◽  
Vol 11 (12) ◽  
pp. 1865 ◽  
Author(s):  
Kenya Kamimura ◽  
Takeshi Yokoo ◽  
Hiroyuki Abe ◽  
Shuji Terai

The liver is a key organ for metabolism, protein synthesis, detoxification, and endocrine function, and among liver diseases, including hepatitis, cirrhosis, malignant tumors, and congenital disease, liver cancer is one of the leading causes of cancer-related deaths worldwide. Conventional therapeutic options such as embolization and chemotherapy are not effective against advanced-stage liver cancer; therefore, continuous efforts focus on the development of novel therapeutic options, including molecular targeted agents and gene therapy. In this review, we will summarize the progress toward the development of gene therapies for liver cancer, with an emphasis on recent clinical trials and preclinical studies.


2020 ◽  
Vol 9 (2) ◽  
pp. 289 ◽  
Author(s):  
Monica Borgatti ◽  
Emiliano Altamura ◽  
Francesca Salvatori ◽  
Elisabetta D’Aversa ◽  
Nicola Altamura

Several types of thalassemia (including β039-thalassemia) are caused by nonsense mutations in genes controlling globin production, leading to premature translation termination and mRNA destabilization mediated by the nonsense mediated mRNA decay. Drugs (for instance, aminoglycosides) can be designed to suppress premature translation termination by inducing readthrough (or nonsense suppression) at the premature termination codon. These findings have introduced new hopes for the development of a pharmacologic approach to cure this genetic disease. In the present review, we first summarize the principle and current status of the chemical relief for the expression of functional proteins from genes otherwise unfruitful for the presence of nonsense mutations. Second, we compare data available on readthrough molecules for β0-thalassemia. The examples reported in the review strongly suggest that ribosomal readthrough should be considered as a therapeutic approach for the treatment of β0-thalassemia caused by nonsense mutations. Concluding, the discovery of molecules, exhibiting the property of inducing β-globin, such as readthrough compounds, is of great interest and represents a hope for several patients, whose survival will depend on the possible use of drugs rendering blood transfusion and chelation therapy unnecessary.


2014 ◽  
Vol 15 (5) ◽  
pp. 7500-7512 ◽  
Author(s):  
Philipp Uhl ◽  
Gert Fricker ◽  
Uwe Haberkorn ◽  
Walter Mier

2012 ◽  
Vol 1 (3) ◽  
pp. 299-305
Author(s):  
Ahmed M. El-Gohary ◽  
Fadia M. Attia ◽  
Fawzy A. Khalil

2011 ◽  
Vol 1 (3) ◽  
pp. 299-305
Author(s):  
Ahmed M. El-Gohary ◽  
Fadia M. Attia ◽  
Fawzy A. Khalil

Kanzo ◽  
2015 ◽  
Vol 56 (7) ◽  
pp. 366-368 ◽  
Author(s):  
Yoshinori Horie ◽  
Hirotoshi Ebinuma ◽  
Masahiro Kikuchi ◽  
Nobuhiro Nakamoto ◽  
Takanori Kanai

Kanzo ◽  
2017 ◽  
Vol 58 (9) ◽  
pp. 486-493 ◽  
Author(s):  
Ryoko Sumi ◽  
Kazuto Fukuda ◽  
Keiko Irishio ◽  
Michiyo Hattori ◽  
Yoshiyuki Sawai ◽  
...  

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