Results 131 to 140 of about 110,488 (256)

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Adeno-associated virus-mediated neuroglobin overexpression ameliorates the N-methyl-N-nitrosourea-induced retinal impairments: a novel therapeutic strategy against photoreceptor degeneration

Therapeutics and Clinical Risk Management, 2017
Ye Tao,1,* Zhen Yang,2,* Wei Fang,2 Zhao Ma,3 Yi Fei Huang,1 Zhengwei Li4 1Department of Ophthalmology, Key Lab of Ophthalmology and Visual Science, Chinese PLA General Hospital, Beijing, 2Department of Neurosurgery, Institute for Functional Brain ...
Tao Y, Yang Z, Fang W, Ma Z, Huang YF, Li Z   +5 more
doaj  

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source

A Mechanistic Model of rAAV Production in Synthetic Cell Lines

Biotechnology and Bioengineering, EarlyView.
ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan, Han‐Jung Kuo, Yu‐Chieh Lin, Yen‐An Lu, Wei‐Shou Hu, Qi Zhang   +5 more
wiley   +1 more source

Effectiveness of resveratrol in inducing adeno-associated virus as a potential definitive therapy for SCN5A mutation in Brugada syndrome: a narrative review

The Egyptian Heart Journal
Objectives Brugada syndrome (BrS) is a hereditary channelopathy that affects cardiac electrical signal transmission, with SCN5A gene mutation being the most common cause.
Andin Zahrani Pateda, Andi Alika Azzahra, Kuni Zakiyyah Sumargo, Muchtar Nora Ismail Siregar   +3 more
doaj   +1 more source

Serotonin 5‐HT7 receptor signaling in neuropsychiatric disorders

Bulletin of the Korean Chemical Society, EarlyView.
5‐HT7R recruits Gs, G12, and β‐arrestin signaling to regulate neuronal plasticity, circuit function, and kinase‐linked intracellular responses. This review summarizes how these pathway‐selective modules contribute to autism spectrum disorder, depression, and schizophrenia, highlighting 5‐HT7R as a pathway‐informed therapeutic target. Abstract Serotonin
Eunseo Park, Hyunah Choo
wiley   +1 more source

Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks

Chemistry – A European Journal, EarlyView.
A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan, Ethan J. French, Emerenziana Runco, Chetan B. Dhakan, Jinwu Yan, Hiroshi Nakashima, Michael T. McMahon, Assaf A. Gilad, Christian T. Farrar   +8 more
wiley   +1 more source

TULP4, a novel E3 ligase gene, participates in neuronal migration as a candidate in schizophrenia

CNS Neuroscience &Therapeutics, EarlyView., 2023
Mutations identified from four SCZ pedigrees resulted in decreased TULP4 expression. Tulp4 knockdown caused delayed neuron migration in embryonic mice, and impaired cognition and prepulse inhibition in adult mice. These phenotypes may be related to TULP4 through its involvement in the formation of a novel E3 ubiquitin ligases.
Yan Bi, Decheng Ren, Fan Yuan, Zhou Zhang, Daizhan Zhou, Xin Yi, Lei Ji, Keyi Li, Fengping Yang, Xi Wu, Xingwang Li, Yifeng Xu, Yun Liu, Peng Wang, Changqun Cai, Chuanxin Liu, Qian Ma, Lin He, Yi Shi, Guang He   +19 more
wiley   +1 more source

Epilepsy characteristics in patients with muscle‐eye‐brain disease: A systematic review of electroclinical features

Epileptic Disorders, EarlyView.
Abstract Background and Objectives Muscle‐Eye‐Brain disease (MEB) is a dystroglycanopathy that belongs to the congenital muscular dystrophies. Central nervous system manifestations include congenital brain abnormalities, neurodevelopmental delay, and epilepsy, making it a rare but important cause of developmental and epileptic encephalopathy.
Stefania Kalampokini, Evripidis Pityrigkas, Eleni Liouta, Georgia Pepe, Vasiliki Poulidou, Martha Spilioti, Vasilios K. Kimiskidis   +6 more
wiley   +1 more source

Inherited metabolic epilepsies–established diseases, new approaches

Epilepsia Open, EarlyView.
Abstract Inherited metabolic epilepsies (IMEs) represent the inherited metabolic disorders (IMDs) in which epilepsy is a prevailing component, often determining other neurodevelopmental outcomes associated with the disorder. The different metabolic pathways affected by individual IMEs are the basis of their rarity and heterogeneity.
Itay Tokatly Latzer, Phillip L. Pearl
wiley   +1 more source