Results 111 to 120 of about 248,807 (309)

CRISPR/Cas9-induced (CTG⋅CAG)n repeat instability in the myotonic dystrophy type 1 locus: implications for therapeutic genome editing [PDF]

, 2017
Myotonic dystrophy type 1 (DM1) is caused by (CTG⋅CAG)n-repeat expansion within the DMPK gene and thought to be mediated by a toxic RNA gain of function.
André, Laurène M., Cumming, Sarah A., Furling, Denis, Gourdon, Geneviève, Monckton, Darren G., Mouly, Vincent, van Agtmaal, Ellen L., van den Broek, Walther J.A.A., van Kessel, Ingeborg D.G., Wansink, Derick G., Wieringa, Bé, Willemse, Marieke   +11 more
core   +4 more sources

CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes

Scientific Reports, 2017
The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 system revolutionized the field of gene editing but viral delivery of the CRISPR/Cas9 system has not been fully explored.
Eric Ehrke-Schulz, Maren Schiwon, Theo Leitner, Stephan Dávid, Thorsten Bergmann, Jing Liu, Anja Ehrhardt   +6 more
doaj   +1 more source

CRISPR/Cas9

Drug Discovery Today, 2019
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR associated nuclease 9 (Cas9), as a powerful genome-editing tool, has revolutionized genetic engineering. It is widely used to investigate the molecular basis of different cancer types. In this review, we present an overview of recent studies in which CRISPR/Cas9 has been used for
Liu, Bin, Saber, Ali, Haisma, Hidde J
openaire   +1 more source

Mapping the Cerebral Organoid Landscape: A Systematic Review of Preclinical 3D Models in Neuroscience

Advanced Healthcare Materials, EarlyView.
Cerebral organoids are transforming brain research, yet the field remains fragmented. This comprehensive systematic review maps 738 studies published between 2014 and 2024 to uncover trends, gaps, and opportunities across neuroscience. Introducing OrganoidMap—an interactive, open‐access platform to explore and compare models—this work enables ...
Anna Wolfram, Vanessa Arnold, Maik Wolfram‐Schauerte, Anastassiya Moskalchuk, Caroline Trust, Marie Vontz, Mona Scheurenbrand, Vijayasarathy Sampath‐Kumar, Sogand Ahari, Carlos Romero‐Nieto, Lisa Sevenich   +10 more
wiley   +1 more source

DISC1 is needed for the organization of cellular components at the immunological synapse. [PDF]

, 2019
T-cells carry out their immune functions through receptor-based recognition and adhesion to target cells. The cell-cell junction that is created from this connection is known as the immunological synapse (IS); a cellular structure composed of organized ...
Ramakrishnan, Adarsh
core   +1 more source

Respiratory Organ‐on‐a‐Chip for Disease Modeling: From Architecture to Functional Integration

Advanced Healthcare Materials, EarlyView.
Respiratory organ‐on‐a‐chip (ROC) models capture key mechanical and cellular cues of the human respiratory system, enabling quantitative dissection of disease mechanisms. This review links ROC architectures to disease modeling, functional integration, and commercialization, and proposes a decision framework that aligns model complexity with mechanistic
Jinzhuo Hu, Yongjie Tang, Sidi Liu, Tao Xu, Qian Liu   +4 more
wiley   +1 more source

Methods for Scarless, Selection-Free Generation of Human Cells and Allele-Specific Functional Analysis of Disease-Associated SNPs and Variants of Uncertain Significance. [PDF]

, 2017
With the continued emergence of risk loci from Genome-Wide Association studies and variants of uncertain significance identified from patient sequencing, better methods are required to translate these human genetic findings into improvements in public ...
Carvajal-Carmona, Luis G, Coggins, Nicole B, O'Geen, Henriette, Segal, David J, Stultz, Jacob   +4 more
core   +4 more sources

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

Recapitulating Endochondral Ossification for Bone Repair: From Development to Engineering Strategy

Advanced Healthcare Materials, EarlyView.
This review summarizes the developmental basis of endochondral ossification (ECO) and its applications in bone tissue engineering (BTE). It first outlines the key biological processes and signaling pathways underlying ECO, then discusses biomaterial‐based engineering strategies derived from these principles, and finally highlights future directions for
Yiqi Su, Zihao He, Qianqian Chen, Long Chen, Caiyi Wei, Zijin Zhou, Jianhao Lin, Hui Li, Dan Xing   +8 more
wiley   +1 more source

Live cell imaging of low- and non-repetitive chromosome loci using CRISPR-Cas9. [PDF]

, 2017
Imaging chromatin dynamics is crucial to understand genome organization and its role in transcriptional regulation. Recently, the RNA-guidable feature of CRISPR-Cas9 has been utilized for imaging of chromatin within live cells. However, these methods are
Adli, Mazhar, Bandaria, Jigar, Darzacq, Xavier, Kuscu, Cem, Mir, Mustafa, Parlak, Mahmut, Qin, Peiwu, Singh, Ritambhara, Szlachta, Karol, Yildiz, Ahmet   +9 more
core   +1 more source