Results 161 to 170 of about 36,553 (255)

Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay. [PDF]

Mol Ther Methods Clin Dev
Soth S, Takakura M, Suekawa M, Onishi T, Hirohata K, Hashimoto T, Maruno T, Fukuhara M, Tsunaka Y, Torisu T, Uchiyama S.   +10 more
europepmc   +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

Induction of tauopathy in a mouse model of amyloidosis using intravenous administration of adeno-associated virus vectors expressing human P301L tau. [PDF]

Alzheimers Dement (N Y)
Finneran DJ, Desjarlais T, Henry A, Jackman BM, Gordon MN, Morgan D.   +5 more
europepmc   +1 more source

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII). II. Treatments in more advanced clinical development

Epilepsia, EarlyView.
Abstract This article summarizes data for 13 investigational treatments for which at least preliminary seizure outcome data in patients with epilepsy were reported at the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices held in Madrid, Spain, on May 3–6, 2026.
Meir Bialer, Cecilie Johannessen Landmark, Matthias J. Koepp, Emilio Perucca, Piero Perucca, Torbjörn Tomson, H. Steve White, Elaine Wirrell   +7 more
wiley   +1 more source

Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders. [PDF]

Int J Mol Sci
Daci R, Flotte TR.
europepmc   +1 more source

Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers. [PDF]

J Ophthalmic Vis Res, 2020
Casey GA, Papp KM, MacDonald IM.
europepmc   +1 more source

Anterior cingulate cortex neuron subtypes differentially regulate seizures

Epilepsia, EarlyView.
Abstract Objective This study aimed to investigate the regulatory roles of distinct neuronal subtypes within the anterior cingulate cortex (ACC) in acute seizures and to identify cell type‐specific mechanisms underlying seizure modulation in this region. Methods Acute seizure models were established in mice via pentylenetetrazol injection.
Ziqian Yan, Ting Tang, Kaishan Wang, Bin Fu, Zesheng Li, Mingshan Liu, Hongyi Huang, Yanfeng Yang, Qiang Zheng, Yangmin Zheng, Yongzhi Shan, Yumin Luo, Guoguang Zhao   +12 more
wiley   +1 more source

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors. [PDF]

Sci Rep, 2020
Bertin B, Veron P, Leborgne C, Deschamps JY, Moullec S, Fromes Y, Collaud F, Boutin S, Latournerie V, van Wittenberghe L, Delache B, Le Grand R, Dereuddre-Bosquet N, Benveniste O, Moullier P, Masurier C, Merten O, Mingozzi F.   +17 more
europepmc   +1 more source

Epilepsy‐associated SCN2A‐L1342P mutation drives network hyperexcitability and widespread transcriptomic changes in human cortical organoids

Epilepsia, EarlyView.
Abstract Objective SCN2A pathogenic mutations, such as the recurrent heterozygous Nav1.2‐L1342P, are monogenic causes of epilepsy. In this human‐induced pluripotent stem cell–derived model system, we aim to investigate the molecular and cellular mechanisms underlying SCN2A‐L1342P‐associated pathology. Methods Using a human male induced pluripotent stem
Maria I. Olivero‐Acosta, Morgan Robinson, Zhefu Que, Zaiyang Zhang, Karen V. Salazar‐Salazar, Hope Elizabeth Harlow, Vinayak Shankar, Seoyeon Hong, Muhan Wang, Conrad M. Otterbacher, Hina Kadono, Manasi Halurkar, Harish Kothandaraman, Nadia Atallah Lanman, Trang Nguyen, Kyle Wettschurack, Benjamin Zirkle, Layan Yunis, Ningren Cui, Xiaoling Chen, Jingliang Zhang, Jiaxiang Wu, William C. Skarnes, Chongli Yuan, Feng Guo, Megan Abbott, Yang Yang   +26 more
wiley   +1 more source

A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene. [PDF]

Mol Ther Methods Clin Dev, 2020
Aronson SJ, Bakker RS, Moenis S, van Dijk R, Bortolussi G, Collaud F, Shi X, Duijst S, Ten Bloemendaal L, Ronzitti G, Muro AF, Mingozzi F, Beuers U, Bosma PJ.   +13 more
europepmc   +1 more source