Results 71 to 80 of about 152,507 (240)

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Cardiac‐Derived ECM Microspheres for Enhanced hiPSC‐CMs Maturation

Advanced Functional Materials, EarlyView.
Cardiac extracellular matrix microspheres derived from decellularized porcine heart provide a biomimetic 3D microenvironment for human induced pluripotent stem cell–derived cardiomyocytes (hiPSC‐CMs). This platform supports short‐ and long‐term culture, enhances structural organization, and promotes electrophysiological and functional maturation of ...
Jiazhu Xu, Joel Aboagye, Marcella Edwards, Nick Rogozinski, Yufeng Wen, Angello Huerta Gomez, Zui Pan, Ge Zhang, Huaxiao Yang, Yi Hong   +9 more
wiley   +1 more source

CRISPR/Cas9, a universal tool for genomic engineering

Вавиловский журнал генетики и селекции, 2016
The CRISPR/Cas9 system was initially described as an element of archeal and bacterial immunity, but gained much attention recently for its outstanding ability to be programmed to target any genomic loci through a short 20-nucleotide sgRNA region. Here we
A. V. Smirnov, A. M. Yunusova, V. A. Lukyanchikova, N. R. Battulin   +3 more
doaj   +1 more source

VIVID: A qPCR‐Based Platform for Sensitive and Quantitative In Vivo Tracking of Extracellular Vesicles

Advanced Functional Materials, EarlyView.
This study introduces VIVID (Vesicle In Vivo Identification using DNA), a qPCR‐based platform that tracks PCR‐amplifiable DNA tags loaded in the EVs for accurate and quantifiable EV biodistribution in vivo. ABSTRACT Extracellular vesicles (EVs) represent promising carriers for nucleic acid therapeutics, offering advantages over synthetic nanoparticles ...
Oscar Boyadjian, Talin Ebrahimian, Jonathan Miranda O'Connor, Janusz Rak, Stephanie Lehoux, Maryam Tabrizian   +5 more
wiley   +1 more source

Patient‐Derived 3D Bioprinted Cardiac Organoid Constructs Reveal Key Pathological Features of Duchenne Muscular Dystrophy

Advanced Healthcare Materials, EarlyView.
Patient‐derived cardiac organoids reveal key features of Duchenne muscular dystrophy cardiomyopathy, including apoptosis, oxidative stress, calcium handling defects, and mechanical remodeling. By integrating organoids into alginate–gelatin bioprinted constructs, disease phenotypes are organized into scalable 3D cardiac tissues displaying extracellular ...
Vittoria Marini, Margalida Campaner Socias, Andreas Dimopoulos, Lorenza Rinvenuto, Enrico Pozzo, Diana Stalkopf, Angelo Serafini, Sara Morri, Ashley Wang, Rita La Rovere, Yoke Chin Chai, Sveva Bollini, Geert Bultynck, H. Llewelyn Roderick, Ioannis Papantoniou, Maurilio Sampaolesi   +15 more
wiley   +1 more source

UGI relocation inside Cas9 reduces Cas9 dependent off target effects in cytosine base editors

Scientific Reports
Cytosine base editors (CBEs) achieve precise C-to-T conversions by addition of uracil DNA glycosylase inhibitor (UGI) with Cas9 nickase (nCas9) and cytidine deaminase, and the conventional fusion at the nCas9 carboxyl terminus effectively inhibits uracil
Zehao Shi, Tian-Lin Cheng
doaj   +1 more source

Advanced 3D Platforms for Modeling CNS Neuroinflammation: Cell Integration, Techniques, and Challenges

Advanced Healthcare Materials, EarlyView.
This review summarizes the roles of key central nervous system cell types, the extracellular matrix, and the blood‐brain barrier in neuroinflammation, and their integration into diverse 3D culture systems. It examines major incorporation strategies, including direct co‐culture, hydrogel encapsulation, transwell migration assays, and bioprinting ...
Emmanuelle D. Aiyegbusi, Julia Di Stefano, Dearbhaile Dooley   +2 more
wiley   +1 more source

Development of an RNA aptamer-assisted CRISPR/Cas9 system for efficiently generating and isolating Cas9-free mutants in plant.

PLoS Genetics
The CRISPR/Cas9 gene-editing system is a powerful tool in plant genetic engineering; however, screening for Cas9-free edited plants remains complex and time-consuming.
Sha Liu, Jiuyuan Bai, Bo Zhan, Junyu Yao, Jiayu Zhang, Jia Yi, Mengyue Dong, Qicong Li, Yucheng Shen, Yazhou Chen, Yun Zhao   +10 more
doaj   +1 more source

CRISPR Cas9 in Pancreatic Cancer Research

Frontiers in Cell and Developmental Biology, 2019
Pancreatic cancer is now becoming a common cause of cancer death with no significant change in patient survival over the last 10 years. The main treatment options for pancreatic cancer patients are surgery, radiation therapy and chemotherapy, but there ...
Hai Yang, Peter Bailey, Christian Pilarsky   +2 more
doaj   +1 more source

Kidney Organoids in Drug Development: Integrating Technological Advances and Standardization for Effective Implementation

Advanced Healthcare Materials, EarlyView.
This review examines how emerging enabling technologies enhance the physiological relevance, scalability, and reproducibility of kidney organoids, while advanced analytical approaches support model validation and deepen mechanistic insight into nephrotoxicity.
Helen Kearney, Silvia M. Mihăilă, Lorenzo Moroni, Carlos Mota   +3 more
wiley   +1 more source