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Part of book or chapter of book
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JIMD Reports
Article . 2013
https://doi.org/10.1007/8904_2...
Part of book or chapter of book . 2012 . Peer-reviewed
Data sources: Crossref
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A Novel Double Mutation in the ABCD1 Gene in a Patient with X-linked Adrenoleukodystrophy: Analysis of the Stability and Function of the Mutant ABCD1 Protein

Authors: Masashi, Morita; Junpei, Kobayashi; Kozue, Yamazaki; Kosuke, Kawaguchi; Ayako, Honda; Kenji, Sugai; Nobuyuki, Shimozawa; +2 Authors

A Novel Double Mutation in the ABCD1 Gene in a Patient with X-linked Adrenoleukodystrophy: Analysis of the Stability and Function of the Mutant ABCD1 Protein

Abstract

We diagnosed an adrenomyeloneuropathy (AMN) patient with a double novel missense mutation, c.284C>A (p.A95D) and c.290A>T (p.H97L) in a single ABCD1 allele. In skin fibroblasts from the patient, no ABCD1 protein was detected by immunoblot analysis, and the C24:0 β-oxidation activity was decreased to a level at which the ABCD1 protein was absent. To determine the responsible gene mutation in the patient, we constructed three kinds of mutated ABCD1 gene expression vectors (c.284C>A, c.290A>T or c.284C>A/c.290A>T) and transfected them into CHO cells stably expressing GFP-SKL (CHO/GFP-SKL cells) or CADDS fibroblasts lacking the ABCD1 gene. ABCD1 (p.H97L) displayed the correct peroxisomal localization in CHO/GFP-SKL cells, but ABCD1 (p.A95D) and ABCD1 (p.A95D/p.H97L) were diffuse in the cytosol. Furthermore, ABCD1 (p.H97L) was detected by immunoblot analysis and restored the C24:0 β-oxidation activity in the CADDS fibroblasts, as the wild type ABCD1 did. On the other hand, ABCD1 (p.A95D) and ABCD1 (p.A95D/p.H97L) were not detected and the C24:0 β-oxidation activity was not restored. These results clearly show that c.284C>A is the responsible gene mutation, whereas c.290A>T is a novel polymorphism.

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
13
Top 10%
Top 10%
Average
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