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
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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
Transcriptome Profiling of Phenylalanine-Treated Human Neuronal Model: Spotlight on Neurite Impairment and Synaptic Connectivity
Sign Up to like & getrecommendations! Published in 2024 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms251810019
Abstract: Phenylketonuria (PKU) is the most common inherited disorder of amino acid metabolism, characterized by high levels of phenylalanine (Phe) in the blood and brain, leading to cognitive impairment without treatment. Nevertheless, Phe-mediated brain dysfunction is… read more here.
Keywords: neuronal model; neurite impairment; human neuronal; phe ... See more keywords