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A Comprehensive Atlas of AAV Tropism in the Mouse.

Mol Ther

January 2025

Department of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA. Electronic address:

Gene therapy with Adeno-Associated Virus (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of ten naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10 and AAVrh74) following systemic delivery into male and female mice. A transgene expressing ZsGreen and Cre recombinase was used to identify transduction in a cell-dependent manner based on fluorescence.

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Does Clostridium Perfringens Epsilon Toxin Mimic an Auto-Antigen Involved in Multiple Sclerosis?

Toxins (Basel)

January 2025

Unité des Toxines Bactériennes, Institut Pasteur, Université Paris Cité, CNRS UMR 2001 INSERM U1306, 75015 Paris, France.

Multiple sclerosis (MS) is a chronic immune-mediated neurological disorder, characterized by progressive demyelination and neuronal cell loss in the central nervous system. Many possible causes of MS have been proposed, including genetic factors, environmental triggers, and infectious agents. Recently, epsilon toxin (ETX) has been incriminated in MS, based initially on the isolation of the bacteria from a MS patient, combined with an immunoreactivity to ETX.

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Temporal lobe epilepsy (TLE) represents a prevalent form of focal epilepsy that often requires surgical intervention and can be resistant to antiseizure medications. Its epidemiology varies across regions due to diagnostic challenges and underestimation of individual neurological traits. Despite these complexities, TLE accounts for a significant proportion of total epilepsies worldwide.

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Type A GABA (γ-aminobutyric acid) receptors (GABA receptors) mediate most fast inhibitory signalling in the brain and are targets for drugs that treat epilepsy, anxiety, depression and insomnia and for anaesthetics. These receptors comprise a complex array of 19 related subunits, which form pentameric ligand-gated ion channels. The composition and structure of native GABA receptors in the human brain have been inferred from subunit localization in tissue, functional measurements and structural analysis from recombinant expression and in mice.

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Top Ten epilepsy research priorities: A UK priority setting partnership.

Seizure

December 2024

Translational and Clinical Research Institute, Newcastle University, Newcastle, United Kingdom; Royal Victoria Infirmary, Newcastle Hospitals, NHS Foundation Trust, Newcastle Upon Tyne, United Kingdom.

Purpose: Research into epilepsy has experienced decades of chronic underfunding compared to other neurological conditions despite its prevalence and seriousness. To evidence the need for greater investment, the Epilepsy Research Institute (formerly Epilepsy Research UK) funded, led and managed a James Lind Alliance (JLA) Priority Setting Partnership (PSP). This "industry standard" methodology brings together healthcare professionals, patients, carers and patient group representatives to identify and prioritise research uncertainties within a defined area of health or care.

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