Adrenomyeloneuropathy (AMN)
This article reviews preclinical data supporting the efficacy of SBT101 gene therapy as a treatment for Adrenomyeloneuropathy (AMN). This neurological condition refers to the loss of neurons -or their function- of the central nervous system affecting, especially the spinal cord and other organs such as the adrenal glands. This occurs due to the expression of an impaired gene called ABCD1 which leads to the production of mutant proteins. There are no yet confirmed cures for this condition but SBT101 could act as a potential gene therapy. Gene therapy includes targeted changes in the genes (structures carrying the genetic information of an organism) to treat, delay or stop a disease. Specifically, SBT101 gene therapy acts by increasing the healthy ABCD1 gene expression, showing a dose-dependent improvement of the condition. Preclinical data also revealed the absence of adverse effects without affecting the rest of the organism’s tissues. As a result, SBT101 gene therapy could be an effective and safe treatment used for patients suffering from AMN without the risk of adverse effects. The severity of the disease’s symptoms can be therefore delayed, allowing a better life quality for the patients diagnosed with AMN.