Targeted Brain Delivery of Dendrimer-4-Phenylbutyrate Ameliorates Neurological Deficits in a Long-Term ABCD1-Deficient Mouse Model of X-Linked Adrenoleukodystrophy.

Although Abcd1 KO mice showed decreased numbers of Smi32 + neurons which was slightly elevated by treatment with D-4PBA, these findings failed to reach significance (Fig. 5 B, C).

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BiP, an ER chaperone and regulator of ER stress, which is induced in response to ER stress, showed a trend towards increased levels in Abcd1 KO vehicle-treated mice, and a slight reduction in KO mice treated with D-4PBA with early treatment (Fig. 4 D).

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