Results 111 to 120 of about 254,775 (306)

CRISPR/Cas9 and Genome Editing in Drosophila

Journal of Genetics and Genomics, 2014
Recent advances in our ability to design DNA binding factors with specificity for desired sequences have resulted in a revolution in genetic engineering, enabling directed changes to the genome to be made relatively easily. Traditional techniques for generating genetic mutations in most organisms have relied on selection from large pools of randomly ...
Bassett, Andrew R., Liu, Ji-Long
openaire   +3 more sources

Developmental and Epileptic Encephalopathy due to Biallelic Pathogenic Variants in PIGM

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective PIGM encodes a critical enzyme in the glycosylphosphatidylinositol (GPI)‐anchor biosynthesis pathway. While promoter‐region mutations in PIGM have been associated with a relatively mild phenotype characterized by portal vein thrombosis and absence seizures, recent evidence suggests that coding‐region mutations result in a more severe
Júlia Sala‐Coromina, Anna Marcé‐Grau, Barbara Masotto, Marta Codina, Lamia BenJemaa, Yasmina Elaribi, Mónica Martinez‐Gallo, Roger Colobran, Angel Sanchez‐Montañez, Irene Valenzuela, Yoshiko Murakami, Alfons Macaya   +11 more
wiley   +1 more source

CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles

Molecular Therapy: Nucleic Acids, 2016
Owing to its easy-to-use and multiplexing nature, the genome editing tool CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats (CRISPR) associated nuclease 9) is revolutionizing many areas of medical research and one of the most amazing
Wei-Jing Dai, Li-Yao Zhu, Zhong-Yi Yan, Yong Xu, Qi-Long Wang, Xiao-Jie Lu   +5 more
doaj   +1 more source

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss‐of‐function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from ...
Aiswarya Saravanan, Jordan Safran, Anna Loughman, Grace Lin, Shivanshi Vaid, Dana Allababidi, Peter M. Tessier, Louis T. Dang   +7 more
wiley   +1 more source

Reshuffling yeast chromosomes with CRISPR/Cas9

PLOS Genetics, 2019
Genome engineering is a powerful approach to study how chromosomal architecture impacts phenotypes. However, quantifying the fitness impact of translocations independently from the confounding effect of base substitutions has so far remained challenging.
Aubin Fleiss, Samuel O'Donnell, Téo Fournier, Wenqing Lu, Nicolas Agier, Stéphane Delmas, Joseph Schacherer, Gilles Fischer   +7 more
openaire   +5 more sources

Electrostatically‐Stabilized PEG‐Free Lipid Nanoparticles for Systemic Nucleic Acid Delivery

Advanced Functional Materials, EarlyView.
This work describes non‐PEGylated layered lipid nanoparticles (nonPEG LLNPs), a gene delivery platform that improves upon standard PEGylated LNPs via electrostatic adsorption of charged polymers. nonPEG LLNPs maintain colloidal stability under biological stresses, enhance circulation time, and mitigate accelerated blood clearance and hepatic ...
Namita Nabar, Deeksha Kumaresh, Eduardo Nombera Bueno, Mae Pryor, Paula T. Hammond   +4 more
wiley   +1 more source

Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements

Nature Biotechnology, 2018
CRISPR–Cas9 is poised to become the gene editing tool of choice in clinical contexts. Thus far, exploration of Cas9-induced genetic alterations has been limited to the immediate vicinity of the target site and distal off-target sequences, leading to the ...
M. Kosicki, Kärt Tomberg, A. Bradley
semanticscholar   +1 more source

Microfluidic Nano‐Assembly of Red‐Blood‐Cell (RBC) Lipids and Components for Engineering Extracellular Vesicles

Advanced Healthcare Materials, EarlyView.
Engineered red blood cell‐derived extracellular vesicles (eRBCEVs) are synthesized via controlled microfluidic assembly from native RBC lipids, enabling tunable encapsulation of proteins, nucleic acids, nanoparticles, and viral vectors. The platform demonstrates reproducible nanoscale architecture, preserved membrane composition, and functional cargo ...
Chiranth K. Nagaraj, Xilal Y. Rima, Kim T. Nguyen, Courtney Culkins, Nana Boateng, Jacob Doon‐Ralls, Alejandro Bresolin, Xin Huang, Hong Li, Vahedi Amid, Ajeet Pal Singh, Dharti Shantaram, Anastasiia Amari, Mangesh D. Hade, Nicholas R. Merriam, Zachary D. Schultz, Scott Q. Harper, Nizar Y. Saad, Setty M. Magaña, Willa A. Hsueh, Rachel E. Kopec, Blaise R. Kimmel, Andre F. Palmer, Eduardo Reátegui   +23 more
wiley   +1 more source

Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine

Genes and Diseases
CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced short palindromic repeats) as a bacterial immune system and plays as a gene ...
Mohadeseh Khoshandam, Hossein Soltaninejad, Marziyeh Mousazadeh, Amir Ali Hamidieh, Saman Hosseinkhani   +4 more
doaj   +1 more source

Efficient DNA-free genome editing of bread wheat using CRISPR/Cas9 ribonucleoprotein complexes

Nature Communications, 2017
Substantial efforts are being made to optimize the CRISPR/Cas9 system for precision crop breeding. The avoidance of transgene integration and reduction of off-target mutations are the most important targets for optimization.
Zhen Liang, Kunling Chen, Tingdong Li, Yi Zhang, Yanpeng Wang, Qian Zhao, Jinxing Liu, Huawei Zhang, Cuimin Liu, Y. Ran, Caixia Gao   +10 more
semanticscholar   +1 more source