Results 41 to 50 of about 194,133 (208)
Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
Theranostics, 2021 CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years, however, the translation of this biotechnology into therapy has been hindered by efficient delivery of CRISPR/Cas9 materials into target cells. Direct delivery of CRISPR/
Song Zhang +3 more
semanticscholar +1 more source
Nature Communications, 2020 CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic targets cannot currently be accessed due to the lack of carriers that can deliver RNPs ...
Tuo Wei +4 more
semanticscholar +1 more source
Science Advances, 2022 We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling, noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe strategy for glioblastoma gene therapy.
Yan Zou +14 more
semanticscholar +1 more source
Interpretable neural architecture search and transfer learning for understanding CRISPR/Cas9 off-target enzymatic reactions [PDF]
arXiv, 2023 Finely-tuned enzymatic pathways control cellular processes, and their dysregulation can lead to disease. Creating predictive and interpretable models for these pathways is challenging because of the complexity of the pathways and of the cellular and genomic contexts. Here we introduce Elektrum, a deep learning framework which addresses these challenges
arxiv
Nature Genetics, 2017 The CRISPR–Cas9 system has revolutionized gene editing both at single genes and in multiplexed loss-of-function screens, thus enabling precise genome-scale identification of genes essential for proliferation and survival of cancer cells.
R. Meyers +29 more
semanticscholar +1 more source
Recovering a Molecule's 3D Dynamics from Liquid-phase Electron Microscopy Movies [PDF]
arXiv, 2023 The dynamics of biomolecules are crucial for our understanding of their functioning in living systems. However, current 3D imaging techniques, such as cryogenic electron microscopy (cryo-EM), require freezing the sample, which limits the observation of their conformational changes in real time.
arxiv
Science Advances, 2022 Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic ...
J. Han +18 more
semanticscholar +1 more source
Evolved Cas9 variants with broad PAM compatibility and high DNA specificity
Nature, 2018 A key limitation of the use of the CRISPR–Cas9 system for genome editing and other applications is the requirement that a protospacer adjacent motif (PAM) be present at the target site. For the most commonly used Cas9 from Streptococcus pyogenes (SpCas9),
J. H. Hu +10 more
semanticscholar +1 more source
CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens
Nucleic Acids Res., 2018 CRISPOR.org is a web tool for genome editing experiments with the CRISPR–Cas9 system. It finds guide RNAs in an input sequence and ranks them according to different scores that evaluate potential off-targets in the genome of interest and predict on ...
J. Concordet, M. Haeussler
semanticscholar +1 more source
Вавиловский журнал генетики и селекции, 2019 The modifcation of crop genomes employing functional components of the microbial CRISPR/Cas immune system is a rapidly developing area of applied research.
S. V. Gerasimova +11 more
doaj +1 more source