Results 81 to 90 of about 396,665 (375)

Engineering CRISPR System‐Based Bacterial Outer Membrane Vesicle Potentiates T Cell Immunity for Enhanced Cancer Immunotherapy

Advanced Materials, EarlyView.
This study develops a multifunctional CRISPR‐dCas9‐based OMV platform termed OMV‐C9I12, which facilitates the coexpression of CXCL9 and IL‐12 within tumor cells. This platform enhances T cell recruitment and activation, synergizes with anti‐PD‐1/PD‐L1 immunotherapy, and amplifies antitumor T cell immunity.
Hongjin Wang, Hengji Zhan, Bolin Pan, Leli Zeng, Zehua Chen, Sen Liu, Qiang Zhang, Xuwei Hong, Junlin Lu, Xinrou Lin, Xiao Zhao, Jiajian Lai, Kaiwen Jie, Ye Li, Jianmei Zhong, Shengmeng Peng, Siting Chen, Changhao Chen, Wenlong Zhong, Shaoxu Wu, Yihang Pan, Tianxin Lin, Xu Chen   +22 more
wiley   +1 more source

Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks [PDF]

, 2003
Gene targeting is the in situ manipulation of the sequence of an endogenous gene by the introduction of homologous exogenous DNA. Presently, the rate of gene targeting is too low for it to be broadly used in mammalian somatic cell genetics or to cure ...
Baltimore, David, Cathomen, Toni, Porteus, Matthew H., Weitzman, Matthew D.   +3 more
core  

AAV-based vectors for human diseases modeling in laboratory animals

Frontiers in Medicine
The development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models.
Timur I. Aliev, Dmitry V. Yudkin
doaj   +1 more source

Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.

Blood, 2018
Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate.
Wolfgang A. Miesbach, K. Meijer, M. Coppens, P. Kampmann, R. Klamroth, R. Schutgens, M. Tangelder, G. Castaman, J. Schwäble, H. Bonig, E. Seifried, F. Cattaneo, C. Meyer, F. Leebeek   +13 more
semanticscholar   +1 more source

Genetic Engineering Methods in Primary T Cells

Advanced Therapeutics, EarlyView.
Primary T cells can be engineered to confer them with novel therapeutic functions, allowing them to treat a variety of conditions. Genetic engineering can be either stable or transient, aiming to either express or inhibit a target gene. This review discusses the various genetic engineering tools available as well as their characteristics and ...
Anthony Youssef, Hui‐Shan Li
wiley   +1 more source

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

Science Translational Medicine, 2019
Adeno-associated viral vectors reconstitute large proteins in the retina via intein-mediated protein trans-splicing. Leveraging naturally occurring splicing for gene therapy Retinal gene therapy using adeno-associated viral (AAV) vectors has been shown ...
P. Tornabene, I. Trapani, R. Minopoli, M. Centrulo, M. Lupo, Sonia de Simone, P. Tiberi, Fabio Dell'aquila, E. Marrocco, C. Iodice, A. Iuliano, C. Gesualdo, S. Rossi, L. Giaquinto, Silvia Albert, C. Hoyng, E. Polishchuk, F. Cremers, E. Surace, F. Simonelli, M. D. De Matteis, R. Polishchuk, A. Auricchio   +22 more
semanticscholar   +1 more source

Lecithin Alleviates Memory Deficits and Muscle Attenuation in Chinese Older Adults and SAMP8 Mice

Advanced Science, EarlyView.
This study opens a new avenue for safeguarding cognition and muscle health, averting disability in older age, and treating age‐related pathologies through lecithin supplementation. It serves as a promising nonpharmacological intervention for the crosstalk of muscle and cognition.
Xianyun Wang, Dajun Li, Xiao Ying Li, Weizhao Lu, Huini Ding, Chengyan Qi, Xuan Wang, Jing Shen, Yafei Chi, Tiantian Li, Michelle M. Dunk, Yu An, Hongmei Huang, Kang Yu, Weili Xu, Rong Xiao, Yuandi Xi   +16 more
wiley   +1 more source

ApoE gene therapy: an overview and update [PDF]

, 2005
Atherosclerosis remains the leading cause of death in industrialized societies. Apolipoprotein E (ApoE) is an attractive candidate to treat hypercholesterolemia and coronary heart disease, as it is a circulating protein with pleiotropic ...
Owen, J.S.
core  

Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights

Viruses
Today, adeno-associated virus (AAV)-based vectors are arguably the most promising in vivo gene delivery vehicles for durable therapeutic gene expression. Advances in molecular engineering, high-throughput screening platforms, and computational techniques
Estrella Lopez-Gordo, Kyle Chamberlain, Jalish Mahmud Riyad, Erik Kohlbrenner, Thomas Weber   +4 more
doaj   +1 more source

Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

JIMD Reports, 2023
Late‐onset Pompe disease (LOPD) is a multisystem disorder with significant myopathy. The standard treatment is enzyme replacement therapy (ERT), a therapy that is lifesaving, yet with limitations.
William B. Hannah, Laura E. Case, Edward C. Smith, Crista Walters, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl   +6 more
doaj   +1 more source