CRISPR/Cas9-mediated knockout of Abcd1 and Abcd2 genes in BV-2 cells: novel microglial models for X-linked Adrenoleukodystrophy

Raas, Quentin; Gondcaille, Catherine; Hamon, Yannick; Leoni, Valerio; Caccia, Claudio; Ménétrier, Franck; Lizard, Gérard; Trompier, Doriane; Savary, Stéphane

doi:10.1016/j.bbalip.2019.02.006

Cited by 34 publications

(47 citation statements)

References 65 publications

(86 reference statements)

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“…Recently a microglial model for ALD was produced by knocking out Abcd1 and Abcd2 in BV‐2 cells, murine microglial cells. These cells accumulate VLCFA and have lipid droplets and striated and whorled lipid inclusions (Raas et al, ).…”

Section: Experimental Modelsmentioning

confidence: 99%

X‐linked adrenoleukodystrophy: Pathology, pathophysiology, diagnostic testing, newborn screening and therapies

Turk

Theda

Fatemi

et al. 2020

Intl J of Devlp Neuroscience

131

113

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Adrenoleukodystrophy (ALD) is a rare X‐linked disease caused by a mutation of the peroxisomal ABCD1 gene. This review summarizes our current understanding of the pathogenic cell‐ and tissue‐specific roles of lipid species in the context of experimental therapeutic strategies and provides an overview of critical historical developments, therapeutic trials and the advent of newborn screening in the USA. In ALD, very long‐chain fatty acid (VLCFA) chain length‐dependent dysregulation of endoplasmic reticulum stress and mitochondrial radical generating systems inducing cell death pathways has been shown, providing the rationale for therapeutic moiety‐specific VLCFA reduction and antioxidant strategies. The continuing increase in newborn screening programs and promising results from ongoing and recent therapeutic investigations provide hope for ALD.

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Section: Experimental Modelsmentioning

confidence: 99%

X‐linked adrenoleukodystrophy: Pathology, pathophysiology, diagnostic testing, newborn screening and therapies

Turk

Theda

Fatemi

et al. 2020

Intl J of Devlp Neuroscience

131

113

View full text Add to dashboard Cite

show abstract

“…It is well known that phagocytosis is affected by lipid membrane composition and is dependent on the extracellular and intracellular lipid environment. Peroxisome lipid metabolism has an extensive impact on lipid membrane composition, and peroxisome dysfunction leads to an unbalanced pool of cellular lipids necessary to support changes in immune cell membranes, which in turn affect the phagocytic capacity of a cell [ 10 , 47 – 49 ]. Membrane properties are indeed modified by changes in their fatty acid and cholesterol content [ 50 ].…”

Section: Peroxisomes Turn On/off Pivotal Cellular Immune Signalingmentioning

confidence: 99%

Peroxisomes in host defense

Cara

2020

PLoS Pathog

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“…One of the hallmarks in these peroxisomal rare neurodegenerative diseases and in other common demyelinating disorders is the accompanying oxidative damage and neuroinflammation [3]. Compelling data indicates that oxidative stress can activate microglia leading to the overproduction of pro-inflammatory molecules [4,5]. Thus, targeting oxidative stress to limit neuroinflammation may open a new pharmacological therapy window for these still incurable devastating peroxisomal diseases.…”

mentioning

confidence: 99%