Developmental and epileptic encephalopathies: from genetic heterogeneity to phenotypic continuum

R Guerrini, V Conti, M Mantegazza… - Physiological …, 2023 - journals.physiology.org
Developmental and epileptic encephalopathies (DEEs) are a heterogeneous group of
disorders characterized by early-onset, often severe epileptic seizures and EEG …

Congenital disorders of glycosylation: Still “hot” in 2020

N Ondruskova, A Cechova, H Hansikova… - … et Biophysica Acta (BBA …, 2021 - Elsevier
Background Congenital disorders of glycosylation (CDG) are inherited metabolic diseases
caused by defects in the genes important for the process of protein and lipid glycosylation …

[HTML][HTML] A quantitative proteome map of the human body

L Jiang, M Wang, S Lin, R Jian, X Li, J Chan, G Dong… - Cell, 2020 - cell.com
Determining protein levels in each tissue and how they compare with RNA levels is
important for understanding human biology and disease as well as regulatory processes …

[HTML][HTML] Transformative network modeling of multi-omics data reveals detailed circuits, key regulators, and potential therapeutics for Alzheimer's disease

M Wang, A Li, M Sekiya, ND Beckmann, X Quan… - Neuron, 2021 - cell.com
To identify the molecular mechanisms and novel therapeutic targets of late-onset
Alzheimer's Disease (LOAD), we performed an integrative network analysis of multi-omics …

Impaired lysosomal acidification triggers iron deficiency and inflammation in vivo

KF Yambire, C Rostosky, T Watanabe, D Pacheu-Grau… - Elife, 2019 - elifesciences.org
Lysosomal acidification is a key feature of healthy cells. Inability to maintain lysosomal acidic
pH is associated with aging and neurodegenerative diseases. However, the mechanisms …

[HTML][HTML] V-ATPase Dysfunction in the Brain: Genetic Insights and Therapeutic Opportunities

A Falace, G Volpedo, M Scala, F Zara, P Striano… - Cells, 2024 - mdpi.com
Vacuolar-type ATPase (v-ATPase) is a multimeric protein complex that regulates H+
transport across membranes and intra-cellular organelle acidification. Catabolic processes …

Presynaptic dysfunction in neurodevelopmental disorders: Insights from the synaptic vesicle life cycle

K Bonnycastle, EC Davenport… - Journal of …, 2021 - Wiley Online Library
The activity‐dependent fusion, retrieval and recycling of synaptic vesicles is essential for the
maintenance of neurotransmission. Until relatively recently it was believed that most …

ATP6V0A1 encoding the a1-subunit of the V0 domain of vacuolar H+-ATPases is essential for brain development in humans and mice

K Aoto, M Kato, T Akita, M Nakashima, H Mutoh… - Nature …, 2021 - nature.com
Abstract Vacuolar H+-ATPases (V-ATPases) transport protons across cellular membranes to
acidify various organelles. ATP6V0A1 encodes the a1-subunit of the V0 domain of V …

Phenotypic and genetic spectrum of ATP6V1A encephalopathy: a disorder of lysosomal homeostasis

R Guerrini, D Mei, K Kerti-Szigeti, S Pepe, MK Koenig… - Brain, 2022 - academic.oup.com
Abstract Vacuolar-type H+-ATPase (V-ATPase) is a multimeric complex present in a variety
of cellular membranes that acts as an ATP-dependent proton pump and plays a key role in …

ATM loss disrupts the autophagy-lysosomal pathway

A Cheng, KH Tse, HM Chow, Y Gan, X Song, F Ma… - Autophagy, 2021 - Taylor & Francis
ABSTRACT ATM (ataxia telangiectasia mutated) protein is found associated with multiple
organelles including synaptic vesicles, endosomes and lysosomes, often in cooperation with …