Article
Dendrimer-N-acetyl-L-cysteine modulates monophagocytic response in adrenoleukodystrophy.
Annals of neurology - 1 Sept 2018
Turk Bela R, Nemeth Christina L, Marx Joel S, Tiffany Carol, Jones Richard, Theisen Benjamin, Kambhampati Siva, Ramireddy Raj, Singh Sarabdeep, Rosen Melissa, Kaufman Miriam L, Murray Connor F, Watkins Paul A, Kannan Sujatha, Kannan Rangaramanujam, Fatemi Ali
Abstract excerpt
OBJECTIVE: X-linked adrenoleukodystrophy (ALD) is a neurodegenerative disorder due to mutations in the peroxisomal very long-chain fatty acyl-CoA transporter, ABCD1, with limited therapeutic options. ALD may manifest in a slowly progressive adrenomyeloneuropathy (AMN) phenotype, or switch to rapid inflammatory demyelinating cerebral disease (cALD), in which microglia have been shown to play a pathophysiological...
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