Results 41 to 50 of about 55,351 (242)

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

Expanding Chemical Space of Nucleic Acid Nanoparticles for Tunable Antiviral‐Like Immunomodulatory Responses and Potent Adjuvant Activity

Advanced Functional Materials, EarlyView.
We introduce a nucleic acid nanoparticle (NANP) platform designed to be rrecognized by the human innate immune system in a regulated manner. By changing chemical composition while maintaining constant architectural parameters, we identify key determinants of immunorecognition enabling the rational design of NANPs with tunable immune activation profiles
Martin Panigaj, Hannah S. Newton, Jian Wang, Laxmi Pandey, Yelixza I. Avilla, Phong Nguyen, Morgan Chandler, Justin Halman, Yasmine Radwan, Stephanie Smith, Elijah Edmondson, Simone Difilippantonio, Chelsea Sanders, Bao Tran, Yongmei Zhao, Shaojun Xie, Edward Cedrone, Barry W. Neun, Meni Wanunu, Nikolay V. Dokholyan, Marina A. Dobrovolskaia, Kirill A. Afonin   +21 more
wiley   +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

Assembling a True “Olympic Gel” From over 16 000 Combinatorial DNA Rings

Advanced Materials, EarlyView.
Olympic gels are an elusive class of soft matter, consisting of molecular networks held together purely by mechanically interlocked rings. Their topological structure promises unique properties and functions, but their synthesis has proven notoriously difficult.
Sarah K. Speed, Yu‐Hsuan Peng, Azra Atabay, Krishna Gupta, Toni Müller, Carolin Fischer, Ilka M. Hermes, Jens‐Uwe Sommer, Michael Lang, Elisha Krieg   +9 more
wiley   +1 more source

Microfluidic Hydroporation Platform for Effective Allogeneic CAR‐T Cell Production and Subsequent Functional Cytotoxicity Analysis

Advanced Materials Technologies, EarlyView.
The hydroporator platform employs controlled hydrodynamic deformation for efficient mRNA and CRISPR/Cas9 delivery into primary human T cells, enabling allogeneic CAR‐T cell manufacturing. It preserves cell functionality and drives potent gene editing, CAR expression, and tumor cytotoxicity, while feature‐based analysis links these functional outcomes ...
Soohyun Jeon, Sein Kim, Hyun‐Young Kim, Duck Cho, Sungsu Park, Seohyun Kim, Aram J. Chung   +6 more
wiley   +1 more source

Cloning and characterization of a wheat homologue of apurinic/apyrimidinic endonuclease Ape1L. [PDF]

PLoS ONE, 2014
Apurinic/apyrimidinic (AP) endonucleases are key DNA repair enzymes involved in the base excision repair (BER) pathway. In BER, an AP endonuclease cleaves DNA at AP sites and 3'-blocking moieties generated by DNA glycosylases and/or oxidative damage.
Botagoz Joldybayeva, Paulina Prorok, Inga R Grin, Dmitry O Zharkov, Alexander A Ishenko, Barbara Tudek, Amangeldy K Bissenbaev, Murat Saparbaev   +7 more
doaj   +1 more source

CypD Dependent mPTP Opening Is Crucial for Oxidized Mitochondrial DNA Release in Ferroptosis

Advanced Science, EarlyView.
Ferroptosis is driven by mitochondrial permeability transition pore (mPTP) opening, which induces mitochondrial swelling and releases oxidized mitochondrial DNA. The released mtDNA activates cGAS–STING signaling, promotes ferritinophagy, and amplifies ferroptotic cell death. Disruption of mtDNA repair sensitizes tumors to ferroptosis in vivo, revealing
Hong Zhou, Wan Fu, Shizuo Liu, Zili Zhang, Hanyan Luo, Qing Zhong   +5 more
wiley   +1 more source

A Cas9 with PAM recognition for adenine dinucleotides

Nature Communications, 2020
Protospacer adjacent motif (PAM) requirements limit the target range of CRISPR endonucleases. Here, the authors graft the 5$$^{\prime}$$ ′ -NAAN-3$$^{\prime}$$ ′ PAM-interacting domain of SmacCas9 onto SpyCas9 to create adenine dinucleotide targeting ...
Pranam Chatterjee, Jooyoung Lee, Lisa Nip, Sabrina R. T. Koseki, Emma Tysinger, Erik J. Sontheimer, Joseph M. Jacobson, Noah Jakimo   +7 more
doaj   +1 more source

Comprehensive Profiling of N6‐methyladnosine (m6A) Readouts Reveals Novel m6A Readers That Regulate Human Embryonic Stem Cell Differentiation

Advanced Science, EarlyView.
This research deciphers the m6A transcriptome by profiling its sites and functional readout effects: from mRNA stability, translation to alternative splicing, across five different cell types. Machine learning model identifies novel m6A‐binding proteins DDX6 and FXR2 and novel m6A reader proteins FUBP3 and L1TD1.
Zhou Huang, Rucong Liu, Zibaguli Wubulikasimu, Wanqing Zhao, Jiaqi Huang, Jiaxuan Wang, Tianyuan Zhang, Rui Fan, Wei Kong, Qinghua Cui, Yang Li, Yuan Zhou   +11 more
wiley   +1 more source

REF Select: Expert System Software for Selecting Restriction Endonucleases for Restriction Endonuclease Fingerprinting

BioTechniques, 1999
REF Select, expert system software, has been developed to assist in the selection of optimal restriction endonucleases for restriction endonuclease fingerprinting (REF), a method for rapid and sensitive mutation screening of long DNA segments (1–2 kb ...
William A. Scaringe, Dongzhou Liao, Qiang Liu, Steve S. Sommer   +3 more
doaj   +1 more source