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Bangari, D. S.

Publications and source records attributed to Bangari, D. S..

2 recordsLinked to original sources

GPR17 modulates oligodendrocyte precursor cell maturation during development but has limited impact on myelin regeneration following demyelinating insults

Pharmacological enhancement of myelin regeneration is broadly recognized as the next frontier in therapeutic approaches for demyelinating diseases of the CNS such as multiple sclerosis. However, although several molecular targets for remyelination have been tested preclinically and in clinical trials, an efficacious and safe myelin repair treatment is yet to be developed. One promising molecular target to enhance myelin repair is the G protein-coupled receptor (GPCR) GPR17, which has been proposed to play a central role in the transition from early oligodendrocyte progenitor cells (OPC) into pre-myelinating oligodendrocytes. These findings are largely supported by studies using transgenic mice where GPR17 deletion results in developmental hypermyelination. Additionally, pharmacological modulation of GPR17 activity has been reported to enhance oligodendrocyte precursor cell (OPC) maturation and myelination. In our studies aimed to characterize and pharmacologically validate GPR17 as a viable target for drug development, we established by means of transcriptional profiling of GPR17 knockout versus wild type OPCs, that absence of this GPCR results in a gene signature revealing minor changes in myelin protein gene expression. Furthermore, blocking GPR17 receptor activity in OPC cultures using selective and potent antagonists or inverse agonists, results in limited enhancement of maturation and myelination in vitro. Importantly, remyelination in both the cuprizone and lysolecithin-induced demyelination models was not enhanced in the absence of GPR17. Our data demonstrate that GPR17 plays a minor role in OPC differentiation during development, and pharmacological modulation of its activity has a marginal effect on oligodendrocyte precursor maturation and myelin regeneration after injury.

neuroscience↗

Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy

Adrenoleukodystrophy (ALD) is a rare neurometabolic disease caused by mutations in the ABCD1 gene, which encodes for the peroxisomal very long chain fatty acid (VLCFAs) transporter. It is a debilitating disorder, which has a spectrum of clinical presentations. The most severe form is a rapidly progressing demyelinating disease called cerebral ALD or CALD. Patients with cALD have a life expectancy of 2-4 years after onset and symptoms often manifest in childhood. The other forms are adrenomyeloneuropathy or AMN, which is a slower progressing degeneration of the spinal cord, and adrenal insufficiency (Addison disease). Since the accumulation of VLCFAs are a common factor in all ALD pathologies, we identified therapeutic approach that could correct this metabolic defect. We developed a substrate reduction therapy (SRT) for ALD in the form of an inhibitor of the lipid elongase principally responsible for the generation of VLCFAs, Elovl1. This small molecule was able to successfully reduce the accumulation of VLCFA in the brain and spinal cord of ABCD1-/y mice. We used single nuclei RNA seq to identify the pathways altered in the ABCD1-/y mouse and corrected with Elovl1 inhibition. Though many lipid metabolism genes and pathways were indeed corrected, treatment with the Elovl1 inhibitor unexpectedly led to profound transcriptional changes beyond correction of pathways altered by loss of ABCD1. These data suggest that Elovl1 inhibition may have broader consequences in ABCD1-/y mice than correction of lipid homeostasis.

pharmacology and toxicology↗