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Re‐analysis of an original CMTX3 family using exome sequencing identifies a known BSCL2 mutation
Journal article   Peer reviewed

Re‐analysis of an original CMTX3 family using exome sequencing identifies a known BSCL2 mutation

Rabia Chaudhry, Aditi Kidambi, Megan Hwa Brewer, Anthony Antonellis, Katherine Mathews, Garth Nicholson and Marina Kennerson
Muscle & nerve, Vol.47(6), pp.922-924
06/2013
DOI: 10.1002/mus.23743
PMCID: PMC5175269
PMID: 23553728
url
https://www.ncbi.nlm.nih.gov/pmc/articles/5175269View
Open Access

Abstract

ABSTRACT Introduction Charcot–Marie–Tooth (CMT) disease is a group of peripheral neuropathies affecting both motor and sensory nerves. CMTX3 is an X‐linked CMT locus, which maps to chromosome Xq26.3–q27.3. Initially, CMTX3 was mapped to a 31.2‐Mb region in 2 American families. We have reexamined 1 of the original families (US‐PED2) by next generation sequencing. Methods Three members of the family underwent exome sequencing. Candidate variants were validated by PCR and Sanger sequencing analysis. Conclusion No pathogenic coding variants localizing to the CMTX3 region were identified. However, exome sequencing identified a known BSCL2 mutation (N88S). This study demonstrates the power of exome sequencing as a tool to identify gene mutations for a small family in the absence of statistically significant linkage data. Muscle Nerve 47: 922–924, 2013
BSCL2 Marie CMTX3 exome sequencing Tooth disease peripheral neuropathy X‐linked Charcot

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