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Ganglioside GM3 is essential for the structural integrity and function of cochlear hair cells
Journal article   Open access   Peer reviewed

Ganglioside GM3 is essential for the structural integrity and function of cochlear hair cells

Misato Yoshikawa, Shinji Go, Shun-ichi Suzuki, Akemi Suzuki, Yukio Katori, Thierry Morlet, Steven M Gottlieb, Michihiro Fujiwara, Katsunori Iwasaki, Kevin A Strauss, …
Human molecular genetics, v 24(10), pp 2796-2807
15 May 2015
PMID: 25652401
url
https://doi.org/10.1093/hmg/ddv041View
Published, Version of Record (VoR) Open

Abstract

Animals Child Child, Preschool Epilepsy - genetics Epilepsy - pathology Epilepsy - physiopathology Female G(M3) Ganglioside - physiology Hair Cells, Auditory - physiology Hair Cells, Auditory - ultrastructure Humans Infant Male Mice Mice, Knockout Mutation, Missense N-Acetylgalactosaminyltransferases - genetics Sialyltransferases - deficiency Sialyltransferases - genetics Stereocilia - ultrastructure
GM3 synthase (ST3GAL5) is the first biosynthetic enzyme of a- and b-series gangliosides. Patients with GM3 synthase deficiency suffer severe neurological disability and deafness. Eight children (ages 4.1 ± 2.3 years) homozygous for ST3GAL5 c.694C>T had no detectable GM3 (a-series) or GD3 (b-series) in plasma. Their auditory function was characterized by the absence of middle ear muscle reflexes, distortion product otoacoustic emissions and cochlear microphonics, as well as abnormal auditory brainstem responses and cortical auditory-evoked potentials. In St3gal5(-/-) mice, stereocilia of outer hair cells showed signs of degeneration as early as postnatal Day 3 (P3); thereafter, blebs devoid of actin or tubulin appeared at the region of vestigial kinocilia, suggesting impaired vesicular trafficking. Stereocilia of St3gal5(-/-) inner hair cells were fused by P17, and protein tyrosine phosphatase receptor Q, normally linked to myosin VI at the tapered base of stereocilia, was maldistributed along the cell membrane. B4galnt1(-/-) (GM2 synthase-deficient) mice expressing only GM3 and GD3 gangliosides had normal auditory structure and function. Thus, GM3-dependent membrane microdomains might be essential for the proper organization and maintenance of stereocilia in auditory hair cells.

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Web of Science research areas
Biochemistry & Molecular Biology
Genetics & Heredity
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