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ANKS1B encoded AIDA-1 regulates social behaviors by controlling oligodendrocyte function
Chang Hoon Cho1,2, Ilana Vasilisa Deyneko1, Dylann Cordova-Martinez1
1Dominick P. Purpura Department of Neuroscience, Albert Einstein College of Medicine, Bronx, NY, USA.
Nature Communications
|December 21, 2023
Summary
ANKS1B neurodevelopmental syndrome (ANDS) involves brain cell (oligodendrocyte) deficits, impacting myelination and social behaviors. Treatment targeting oligodendrocyte maturation may improve social deficits in patients with ANDS.
Area of Science:
- Neuroscience
- Genetics
- Developmental Biology
Background:
- ANKS1B neurodevelopmental syndrome (ANDS) is a rare genetic disorder linked to heterozygous deletions in the ANKS1B gene.
- The ANKS1B gene encodes the AIDA-1 protein, crucial for synaptic plasticity in neurons.
Purpose of the Study:
- To investigate the role of oligodendroglial deficits in the pathophysiology of ANKS1B neurodevelopmental syndrome (ANDS).
- To explore potential therapeutic strategies targeting oligodendrocyte dysfunction in ANDS.
Main Methods:
- Utilized Anks1b-deficient mouse models to study oligodendrocyte development and function.
- Examined the impact of selective Anks1b loss in oligodendrocyte lineage versus neuronal populations.
- Assessed behavioral outcomes, including social preference and sensory reactivity.
- Investigated the therapeutic potential of clemastine, a myelination-enhancing drug.
Main Results:
- Anks1b deficiency in mice led to impaired oligodendrocyte maturation, myelination, and Rac1 function, mirroring white matter abnormalities in ANDS patients.
- Selective loss of Anks1b in oligodendrocytes, not neurons, replicated social and sensory deficits observed in global Anks1b haploinsufficiency.
- Clemastine treatment rescued social preference deficits in adult Anks1b-deficient mice.
Conclusions:
- Oligodendroglial deficits and impaired myelination are key contributors to the pathophysiology of ANDS.
- Abnormal Rac1 activity in oligodendrocytes may underlie the social behavior deficits in ANDS.
- Targeting oligodendrocyte maturation with drugs like clemastine shows promise for treating behavioral symptoms in ANDS.
