Therapeutic approaches for shankopathies

Therapeutic approaches in patients with β-thalassemia

Beta-thalassemia (β-thal) is a congenital hemoglobinopathy explained by a decreased level (β+) or absence (βο) of β-globin gene expression. Microcytic hypochromic anemia and various clinical symptoms comprising severe anemia to clinically nonsymptomatic features. Treatment with an ordered blood transfusion and iron chelator agents can decrease transfusion iron overload that causes normal matura...

Therapeutic approaches for corneal neovascularization

Angiogenesis refers to new blood vessels that originate from pre-existing vascular structures. Corneal neovascularization which can lead to compromised visual acuity occurs in a wide variety of corneal pathologies. A large subset of measures has been advocated to prevent and/or treat corneal neovascularization with varying degrees of success. These approaches include topical corticosteroid admi...

Novel Therapeutic Approaches for Neuroblastoma

Toxin-Based Therapeutic Approaches

Protein toxins confer a defense against predation/grazing or a superior pathogenic competence upon the producing organism. Such toxins have been perfected through evolution in poisonous animals/plants and pathogenic bacteria. Over the past five decades, a lot of effort has been invested in studying their mechanism of action, the way they contribute to pathogenicity and in the development of ant...

Mitochondrial diseases: therapeutic approaches.

Therapy of mitochondrial encephalomyopathies (defined restrictively as defects of the mitochondrial respiratory chain) is woefully inadequate, despite great progress in our understanding of the molecular bases of these disorders. In this review, we consider sequentially several different therapeutic approaches. Palliative therapy is dictated by good medical practice and includes anticonvulsant ...