Phosphatidylserine improves axonal transport by inhibition of HDAC and has potential in treatment of neurodegenerative diseases

Shiran Naftelberg, Gil Ast*, Eran Perlson

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

5 Scopus citations

Abstract

Familial dysautonomia (FD) is a rare children neurodegenerative disease caused due to a point mutation in the IKBKAP gene that results in decreased IKK complex-associated protein (IKAP) protein production. The disease affects mostly the dorsal root ganglion (DRG) and the sympathetic ganglion. Recently, we found that the molecular mechanisms underlying neurodegeneration in FD patients are defects in axonal transport of nerve growth factors and microtubule stability in the DRG. Neurons are highly polarized cells with very long axons. In order to survive and maintain proper function, neurons depend on transport of proteins and other cellular components from the neuronal body along the axons. We further demonstrated that IKAP is necessary for axon maintenance and showed that phosphatidylserine acts as an HDAC6 inhibitor to rescue neuronal function in FD cells. In this review, we will highlight our latest research findings.

Original languageEnglish
Pages (from-to)534-537
Number of pages4
JournalNeural Regeneration Research
Volume12
Issue number4
DOIs
StatePublished - Apr 2017

Funding

FundersFunder number
Israeli National Network of Excellence in Neuroscience1234944, 561/11
S.N.
Dysautonomia Foundation
Teva Pharmaceutical Industries
Seventh Framework Programme309377
European Research Council
Israel Science Foundation142/13, 1439/14

    Keywords

    • Axonal transport
    • Familial dysautonomia
    • HDAC6
    • Microtubule
    • Neurodegeneration
    • Phosphatidylserine

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