Results 171 to 180 of about 116,785 (352)

Rescue of the Adeno-Associated Virus Genome from a Plasmid Vector: Evidence for Rescue by Replication

, 2003
Peter Ward, Per Elias, R. Michael Linden
openalex   +1 more source

Mitochondrial Transplantation as a Therapeutic Strategy for Inherited Mitochondrial Diseases

Advanced Science, EarlyView.
Mitochondrial transplantation (MTx) offers a promising therapeutic avenue for mitochondrial diseases. This review comprehensively evaluates MTx, differentiating its feasibility for mtDNA‐ and nDNA‐based disorders. It examines its potential for genetic correction, alongside inherent limitations, technical challenges, and crucial ethical considerations ...
Parmeshar Singh, Amir Tahavvori, Cyrus E. Kuschner, Blanca B. Espin, Jacob Kazmi, Sofhia V. Ramos, Tai Yin, Kei Hayashida, Kanako Ito‐Hagiwara, Yusuke Endo, Keitaro Yoshioka, Jun Hagiwara, Avijot Sohi, Alisha Oropallo, Ghania Haddad, Timmy Li, Lance B. Becker, Junhwan Kim   +17 more
wiley   +1 more source

A Nonviral Neo‐Nucleocapsid for Cell‐Specific RNA Delivery Developed by Pseudo‐Cyclic Peptide Grafting and Directed Evolution

Angewandte Chemie, EarlyView.
Nonviral neo‐nucleocapsids developed from a repurposed bacterial protein cage, and macrocyclic peptide pharmacophores via protein‐protein ligation, bacteria‐based directed evolution, lasso‐grafting, and mammalian cell‐based directed evolution enabled cell specific RNA delivery.
Daiki Kanayama, Naohiro Terasaka, Kazuki Kato, Sae Okazaki, Hiroaki Suga   +4 more
wiley   +2 more sources

Lactylation Reprogramming in the Bone Infection Microenvironment Identifies PGK1 K361 as a Potential Therapeutic Target for Osteogenic Dysfunction

Advanced Science, EarlyView.
Staphylococcus aureus (S. aureus) infection creates a high‐lactate microenvironment, promoting p300‐mediated lactylation of PGK1 at lysine 361 (K361). Lactylated PGK1 translocates to the mitochondrial outer membrane and interacts with VDAC3. This interaction triggers FtMt downregulation, iron accumulation, and excessive PINK1/Parkin‐mediated mitophagy,
Han‐jun Qin, Si‐ying He, Ting‐hui Xiao, Da‐mao Dai, Xin‐jia Hu, Nan Jiang   +5 more
wiley   +1 more source

A Novel Acetylcholine Nanosensor for Single Vesicle Storage and Sub‐Quantal Exocytosis in Living Neurons and Organoids

Angewandte Chemie, EarlyView.
Schematic representation of M@E@CF nanosensors for detecting vesicular storage and release in cholinergic neurons and brain organoids. (A) Nano‐tip microelectrodes modification via molds fabricated through 3D printing. (B) the reaction mechanism for acetylcholine detection at the electrode interface.
Wanying Zhu, Yufan Zhang, Hanwen Yu, Da Wang, Xinyue Zhang, Xiaowen Du, Shanshan Wu, Bingzhi Li, Yizhou Zhang, Hongliang Xin, Xing Guo, Yan Liu   +11 more
wiley   +2 more sources

Temporal glomerular gene expression dynamics during disease progression in a mouse model of hypertension‐accelerated diabetic kidney disease

Animal Models and Experimental Medicine, EarlyView.
The current study characterized temporal changes in glomerular gene expression, pathology, and biomarkers in a mouse model of hypertension‐accelerated diabetic kidney disease (DKD). Progressive albuminuria and glomerulosclerosis were paralleled by dynamic transcriptomic changes associated withmetabolic dysfunction, extracellular matrix remodeling, and ...
Adam B. Marstrand‐Jørgensen, Frederikke Emilie Sembach, Maria Ougaard, Ditte Hansen, Mette Viberg Østergaard, Henrik H. Hansen, Louise S. Dalbøge, Ole Jørgen Kaasbøll, Michael Christensen   +8 more
wiley   +1 more source

Generating golden Syrian hamsters with conditional alleles via zygote microinjection of CRISPR/Cas9

Animal Models and Experimental Medicine, EarlyView.
We established the first conditional knockout (cKO) model in the golden Syrian hamster by CRISPR/Cas9‐mediated genome editing. Cas9 protein, two sgRNAs, and a donor plasmid carrying loxP‐flanked exon 2 of the ApoF gene were microinjected into one‐cell embryos. The floxed allele was efficiently generated (up to 27%) and transmitted through the germline.
Wei Chen, Xu Zhang, Rui Fan, Xia Li, Feifei Guan, Gefan Wan, Weining Kong, Xiaolong Qi, Shuo Pan, Sijing Shi, Yuanlong Su, Shan Gao, Wei Huang, Xunde Xian, Jiangning Liu, Yuhui Wang, Yuanwu Ma   +16 more
wiley   +1 more source

Co‐Opting MBNL‐Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy

Annals of Neurology, EarlyView.
Objective Myotonic dystrophy type 1 (DM1) is a highly variable, multisystemic genetic disorder caused by a CTG repeat expansion in the 3′ untranslated region of DMPK. Toxicity is exerted by repeat‐containing DMPK transcripts that sequester muscleblind‐like (MBNL) proteins and lead to deleterious yet predictable changes in alternative splicing.
Samuel T. Carrell, Ellie M. Carrell, Ryan Giovenco, Beverly L. Davidson   +3 more
wiley   +1 more source

Molecular Characterization of the Heparin-Dependent Transduction Domain on the Capsid of a Novel Adeno-Associated Virus Isolate, AAV(VR-942)

, 2008
Michael Schmidt, L. Govindasamy, Sandra Afione, Nick Kaludov, Mavis Agbandje‐McKenna, John A. Chiorini   +5 more
openalex   +1 more source

576. Highly Efficient Transgene Expression Using Pseudotyped Adeno Associated Virus Type I in the Mammalian Brain [PDF]

, 2002

openalex   +1 more source