Researchers from University College London and collaborating institutions have recently published results from their study aiming to optimize gene therapy for CLN5 disease.
Korsana Biosciences Inc. has emerged from stealth, with its development of therapeutics to treat neurodegenerative diseases initially focused on Alzheimer’s disease.
Chronic migraine is a neurological disorder characterized by recurrent headache episodes. Recent findings have implicated microglia in the trigeminal nucleus caudalis in chronic migraine-related central sensitization. Activated microglial cells release inflammatory and neurotrophic factors that interact with the neurons involved in the process.
Building on the foundation laid in 2020, researchers at the University of California, San Francisco (UCSF) have now shown that targeting the GPI-anchored vascular enzyme TNAP can reproduce the cognitive benefits previously attributed to the liver-derived exercise factor GLPD1.
The University of California has reported new azocino[4,5,6-cd]indoles acting as 5-HT2A receptor agonists and thus potentially useful for the treatment of neurological and psychiatric disorders.
Nektar Therapeutics Inc. has established an academic research collaboration with the University of California, San Francisco (UCSF) to explore the role of tumor necrosis factor receptor 2 (TNFR2) agonism in models of multiple sclerosis (MS) with the aim of supporting progression of NKTR-0165, Nektar’s first-in-class TNFR2 agonist antibody.
The variety of organoids that can be developed in vitro is enabling major advances. Depending on the type of tissues and the research goals, these small 3D cell-based structures that mimic real tissue offer certain advantages over animal models. Scientists at the University of Padova in Italy have created human neuromuscular organoids to reproduce cancer-induced muscle cachexia, a condition that murine models do not accurately replicate.
Hunter syndrome, also called mucopolysaccharidosis II, is an X-linked genetic lysosomal disorder caused by loss-of-function mutations in the IDS gene, encoding iduronate-2-sulfatase (I2S). I2S is a lysosomal enzyme responsible for the cleavage of glycosaminoglycans (GAGs), and its deficiency results in accumulation of GAGs leading to a multisystemic disorder.
Although activation of the μ-opioid receptor (MOR) provides potent analgesia and remains central to pain management, its agonists are associated with adverse effects such as respiratory depression, tolerance, dependence and pruritus. Bifunctional μ-/κ-opioid receptor (MOR/KOR) agonists aim to retain MOR-driven analgesia while leveraging the antipruritic and complementary analgesic effects of KOR activation to improve overall therapeutic tolerability.
Researchers have explored the effects of human umbilical cord mesenchymal stem cells (hUC-MSCs) on cognitive recovery in anti-N-methyl-D-aspartate receptor (NMDAR) encephalitis, a rare, neuronal antibody-mediated disorder characterized by neuropsychiatric symptoms and impaired cognitive function. hUC-MSCs have previously shown immunomodulatory and anti-inflammatory properties.
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