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Brain-derived neurotrophic factor expression increases after enzyme replacement therapy in Gaucher disease.

Abstract：

:Mutations in the GBA gene are related to an increased risk of developing neurodegenerative diseases. The exact molecular mechanisms involved in the interaction between GBA and α-synuclein, a protein that has been associated with several neurological diseases, remain unsolved. Brain-derived neurotrophic factor (BDNF) is a neurotrophin that is important for the normal development of the peripheral and central nervous system, and it plays a key role in neuronal survival and synaptic plasticity in the adult brain. A reduction in BDNF expression has been reported in patients with Parkinson's disease, Alzheimer's disease and dementia with Lewy bodies. We analyzed BDNF levels in the plasma of Gaucher Disease (GD) patients who were not being treated with enzyme replacement therapy (ERT) and then subsequently following ERT; we compared the levels to those of healthy controls. We demonstrated that BDNF levels were remarkably diminished in GD patients who were under no specific treatment and these levels increased following ERT. This is the first study that demonstrates a variation in the plasma levels of a neurotrophic factor in GD type 1 patients. Further studies are required to correlate BDNF level variations with the clinical findings and the response to therapy in GD patients. Low levels of BDNF are associated with neurodegenerative diseases; therefore, BDNF could provide a new therapeutic target for GD patients with neurological symptoms.

journal_name

J Neuroimmunol

journal_title

Journal of neuroimmunology

authors

Vairo F,Sperb-Ludwig F,Wilke M,Michellin-Tirelli K,Netto C,Neto EC,Doederlein Schwartz IV

doi

10.1016/j.jneuroim.2014.11.005

subject

Has Abstract

pub_date

2015-01-15 00:00:00

pages

190-3

eissn

0165-5728

issn

1872-8421

pii

S0165-5728(14)00951-5

journal_volume

278

pub_type

杂志文章

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