Results 181 to 190 of about 70,372 (263)

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

Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy. [PDF]

Nat Med
Buddle S, Brown LK, Morfopoulou S, Torres Montaguth OE, Scoto M, Herder V, Dhawan A, Brown JR, Atkinson L, Kopec A, Davis D, Storey N, Campos L, Sebire N, Macpherson H, Lee J, Orton R, Baranello G, Asamaphan P, Ilia G, Karda R, Belfield H, ISARIC4C Investigators, Counsell J, Waddington SN, Thomson EC, Muntoni F, Breuer J.   +27 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

Adeno-Associated Virus-Based Gene Therapy for Lafora Disease in <i>Epm2b</i>-Deficient Mice. [PDF]

Int J Mol Sci
Zafra-Puerta L, Iglesias-Cabeza N, Sanz P, García-Gimeno MA, Sánchez-Martín G, Sánchez MP, Serratosa JM.   +6 more
europepmc   +1 more source

Tropism and Retinal Transduction Efficiency of Adeno-Associated Virus Serotypes in Mice. [PDF]

Invest Ophthalmol Vis Sci
Wu W, McRae J, Brown A, Agbaga MP.
europepmc   +1 more source

Reply to a letter from Miesbach and Srivastava: response to hemophilia B gene therapy based on adeno-associated virus serotype 5 antibody status. [PDF]

Res Pract Thromb Haemost
Klamroth R, Monahan PE, Le Quellec S.
europepmc   +1 more source

Suppressing microtubule detyrosination augments adeno-associated virus 2 endosomal escape and gene delivery. [PDF]

J Cell Sci
Tripathi S, Huda S, Kar J, Chandra D, Jayandharan GR, Mohan N.   +5 more
europepmc   +1 more source

Heterogeneity in an adeno-associated virus transfection-based production process limits the production efficiency. [PDF]

Sci Rep
Ladd B, Tunmats S, Gräslund T, Daramola O, Rockberg J, Chotteau V.   +5 more
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