Editorial: Advanced (gene and cell) therapies for central nervous system applications

Mendonça, Liliana; Webster, Christopher; Boltze, Johannes; Nóbrega, Clévio

http://hdl.handle.net/10400.1/19321

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Abstract(s)

Advanced therapies as defined by the European Medicines Agency (EMA) comprises strategies involving gene therapy, cell therapy, and tissue engineering. Overall, these strategies offer a wide range of possibilities to treat and cure diseases, including those affecting the central nervous system (CNS). In this line, Advanced (Gene and Cell) Therapies for Central Nervous System Applications Research Topic was intended to provide a platform for researchers to publish their findings, contributing to the continuous advance of this research area. Nine papers were accepted and published in this Research Topic, from which four described original research data, two papers were reviews and two mini reviews, and one paper focused on hypothesis and theory. In the paper Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques (Arotcarena et al.), presented new data on the biodistribution and CNS transduction efficiencies after lumbar intrathecal bolus delivery of identical doses of either AAV-PH.B:CAG-EGFP or AAV-PHP.eB:CAG-EGFP in rhesus macaque monkeys.

Keywords

Gene augmentation Gene silencinggene editing Viral and non-viral vectors for gene therapy Cell therapycell replacement and neuroprotection Brain experimental models-brain organoids Brain delivery and brain diseases