This preliminary study claim that AMT’s technology may possess overcome among the major complications of shRNA therapies, efficient and non-toxic intracellular delivery specifically, mentioned Jorn Aldag, CEO of Amserdam Molecular Therapeutics. In the study, AMT used its proprietary AAV-structured platform to effectively deliver a shRNA that silences both human and mouse Apolipoprotein B100 . An individual intravenous administration triggered prolonged ApoB100 gene silencing that was sequence-specific and not associated with liver toxicity, oversaturation of the cellular miRNA induction or machinery of immune responses.‘Dr. Acosta includes a very peaceful presence, he truly was heaven sent.’ ‘This is a very hard case, but I sensed we could take action for Laurie,’ said Acosta, who asked J. Patrick Johnson, M.D., co-director of Cedars-Sinai’s Backbone Stem Cell Research Plan, to assist him with both a surgical plan and the procedures. Laurie’s transfer to Cedars-Sinai was arranged, and a united team of specialists – oncologists, rheumatologists and neurologists, among them – mobilized to devise a comprehensive treatment solution. The bone that once guaranteed screws and rods at the back of Laurie’s head have been totally re-absorbed, and equipment from the initial fusion surgery was almost poking through her pores and skin.