...Thomas A. Fox; Claire Booth ABSTRACT Effective gene therapy approaches have been developed for many rare diseases, including inborn errors of immunity and metabolism, haemoglobinopathies and inherited blindness. Despite successful pre-clinical and clinical results, these gene therapies...

...-generation disease models to incorporate these therapeutic developments. Gene therapy Motor neuron disease Spinal muscular atrophy Neuromuscular disease Stichting Spieren voor Spieren http://dx.doi.org/10.13039/501100013428 H2020 Marie Skłodowska-Curie Actions http...

..., especially for those that cannot benefit from HSCT. Here, we review preclinical and proof-of-concept studies, such as gene therapy, systematic administration of deficient protein, in utero HSCT and gene editing. A recent elegant study found that osteoclasts are long-lived syncytia that acquire new nuclei...

... glucose uptake, improving insulin tolerance and ameliorating hepatic steatosis. Therefore, we conclude that a gene therapy approach for hepatic IRA expression could be a safe and promising tool for the regulation of hepatic glucose consumption and lipid metabolism, two key processes in the development...

.... , Barry , M. A. , Kudva , Y. C. and Ikeda , Y. ( 2013 ). Global gene expression profiling of pancreatic islets in mice during streptozotocin-induced β-cell damage and pancreatic Glp-1 gene therapy . Dis. Model. Mech.   6 , 1236 - 1245 . 10.1242/dmm.012591 Tonne , J. M...

... or repair the defective dystrophin gene by gene therapy. Numerous animal models of DMD have been developed over the last 30 years, ranging from invertebrate to large mammalian models. mdx mice are the most commonly employed models in DMD research and have been used to lay the groundwork for DMD gene therapy...