Results 171 to 180 of about 36,553 (255)

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

Applications and Limitations of Equilibrium Density Gradient Analytical Ultracentrifugation for the Quantitative Characterization of Adeno-Associated Virus Vectors. [PDF]

Anal Chem
Hirohata K, Yamaguchi Y, Maruno T, Shibuya R, Torisu T, Onishi T, Chono H, Mineno J, Yuzhe Y, Higashiyama K, Masumi-Koizumi K, Uchida K, Yamamoto T, Uchida E, Okada T, Uchiyama S.   +15 more
europepmc   +1 more source

Precision therapies for genetic epilepsies in 2025: Promises and pitfalls

Epilepsia Open, EarlyView.
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang, Emilio Perucca, Samuel F. Berkovic, Piero Perucca   +3 more
wiley   +1 more source

Adeno-associated virus vectors and neurotoxicity-lessons from preclinical and human studies. [PDF]

Gene Ther
Stone D, Aubert M, Jerome KR.
europepmc   +1 more source

Correction to: Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors, by Westhaus et al. Hum Gene Ther 2023;34(7-8):273-288; doi: 10.1089/hum.2022.188. [PDF]

Hum Gene Ther, 2023
europepmc   +1 more source

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors. [PDF]

Sci Rep, 2020
Karda R, Rahim AA, Wong AMS, Suff N, Diaz JA, Perocheau DP, Tijani M, Ng J, Baruteau J, Martin NP, Hughes M, Delhove JMKM, Counsell JR, Cooper JD, Henckaerts E, Mckay TR, Buckley SMK, Waddington SN.   +17 more
europepmc   +1 more source

Enhancing Optogenetics‐Based Cancer Therapy Via Nanotechnology

Exploration, EarlyView.
Nanotechnology enhances optogenetics‐based cancer therapy through improved delivery systems and non‐invasive optical regulation. It further enables precise and programmable control of optogenetic living therapeutics for localized antitumor responses.
Honggang Shen, Wenzhe Yi, Rongzhang Li, Shuangshuang Hu, Xindi Qian, Dan Yan, Qian Zou, Guanru Wang, Yaping Li   +8 more
wiley   +1 more source

Gene delivery to neurons in the auditory brainstem of barn owls using standard recombinant adeno-associated virus vectors. [PDF]

Curr Res Neurobiol, 2020
Thiele N, Hildebrandt KJ, Köppl C.
europepmc   +1 more source

Strategies and mechanisms of precision genome engineering: From gene editing to genome writing

iMetaOmics, EarlyView.
In this review, we examined the progression of genome manipulation from stochastic nuclease‐mediated cutting toward precise editing and programmable genome writing. We discussed tools like multi‐kilobase RNA‐guided integrators and Artificial Intelligence (AI)‐designed effectors and showed how these advances enable researchers to treat genomes as ...
Kerui Huang, Jianhong Tian, Wenyan Zhao, Siqin Zhang, Lixuan Xiang, Meiqing Yang, Lu Liu, Yazhou Huang, Ningyun Zhang, Lei Sun, Peng Xie, Nanyu Kuang, Hong Zhou, Chengfeng Qiu, Jun Yan, Xiaocheng Jiang, Qiang Tu, Yun Wang, Haoliang Hu, Youming Zhang   +19 more
wiley   +1 more source

Site-Specific Post-translational Surface Modification of Adeno-Associated Virus Vectors Using Leucine Zippers. [PDF]

ACS Synth Biol, 2020
Thadani NN, Yang J, Moyo B, Lee CM, Chen MY, Bao G, Suh J.   +6 more
europepmc   +1 more source