Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia.
Sign Up to like & getrecommendations! Published in 2020 at "Biomaterials science"
DOI: 10.1039/c9bm01757g
Abstract: Increasing frataxin protein levels through gene therapy is envisaged to improve therapeutic outcomes for patients with Friedreich's ataxia (FRDA). A non-viral strategy that uses submicrometer-sized multilayered particles to deliver frataxin-encoding plasmid DNA affords up to… read more here.
Keywords: particle mediated; mediated delivery; friedreich ataxia; frataxin ... See more keywords
Aromatic l-amino acid decarboxylase deficiency: a patient-derived neuronal model for precision therapies
Sign Up to like & getrecommendations! Published in 2021 at "Brain"
DOI: 10.1093/brain/awab123
Abstract: Abstract Aromatic l-amino acid decarboxylase (AADC) deficiency is a complex inherited neurological disorder of monoamine synthesis which results in dopamine and serotonin deficiency. The majority of affected individuals have variable, though often severe cognitive and… read more here.
Keywords: amino acid; neuronal model; deficiency; aromatic amino ... See more keywords