Precision genome editing in the CRISPR era
J Salsman, G Dellaire - Biochemistry and cell biology, 2017 - cdnsciencepub.com
With the introduction of precision genome editing using CRISPR–Cas9 technology, we have
entered a new era of genetic engineering and gene therapy. With RNA-guided …
entered a new era of genetic engineering and gene therapy. With RNA-guided …
rAAV-mediated overexpression of SOX9 and TGF-β via carbon dot-guided vector delivery enhances the biological activities in human bone marrow-derived …
W Meng, A Rey-Rico, M Claudel, G Schmitt… - Nanomaterials, 2020 - mdpi.com
Scaffold-assisted gene therapy is a highly promising tool to treat articular cartilage lesions
upon direct delivery of chondrogenic candidate sequences. The goal of this study was to …
upon direct delivery of chondrogenic candidate sequences. The goal of this study was to …
Frequent loss of heterozygosity in CRISPR-Cas9–edited early human embryos
G Alanis-Lobato, J Zohren… - Proceedings of the …, 2021 - National Acad Sciences
CRISPR-Cas9 genome editing is a promising technique for clinical applications, such as the
correction of disease-associated alleles in somatic cells. The use of this approach has also …
correction of disease-associated alleles in somatic cells. The use of this approach has also …
The potential of CRISPR/Cas9 genome editing for the study and treatment of intervertebral disc pathologies
O Krupkova, E Cambria, L Besse, A Besse… - JOR …, 2018 - Wiley Online Library
The CRISPR/Cas9 system has emerged as a powerful tool for mammalian genome
engineering. In basic and translational intervertebral disc (IVD) research, this technique has …
engineering. In basic and translational intervertebral disc (IVD) research, this technique has …
CRISPR/Cas9 flexes its Muscles: In vivo somatic gene editing for muscular dystrophy
T VandenDriessche, MK Chuah - Molecular Therapy, 2016 - cell.com
© The American Society of Gene & Cell Therapy commentary dality. Both AAV8 and AAV9
mediate efficient gene transfer in skeletal muscle and heart22–24 after systemic …
mediate efficient gene transfer in skeletal muscle and heart22–24 after systemic …
Genome editing for human osteoarthritis–a perspective
D Almarza, M Cucchiarini, J Loughlin - Osteoarthritis and cartilage, 2017 - oarsijournal.com
The ability to efficiently and reproducibly edit genomes using engineered nucleases is one
of the major biological advances of the past few decades and is generating considerable …
of the major biological advances of the past few decades and is generating considerable …
Generation of cell-type-specific gene mutations by expressing the sgRNA of the CRISPR system from the RNA polymerase II promoters
J Wang, X Li, Y Zhao, J Li, Q Zhou, Z Liu - Protein & cell, 2015 - academic.oup.com
Recently, the CRISPR/Cas9 system is emerging as a powerful tool for genome editing
(Chang et al., 2013; Li et al., 2013; Niu et al., 2014; Shen et al., 2013; Wan et al., 2015; …
(Chang et al., 2013; Li et al., 2013; Niu et al., 2014; Shen et al., 2013; Wan et al., 2015; …
Remodeling of human osteochondral defects via rAAV-mediated co-overexpression of TGF-β and IGF-I from implanted human bone marrow-derived mesenchymal …
S Morscheid, JK Venkatesan, A Rey-Rico… - Journal of Clinical …, 2019 - mdpi.com
The application of chondrogenic gene sequences to human bone marrow-derived
mesenchymal stromal cells (hMSCs) is an attractive strategy to activate the reparative …
mesenchymal stromal cells (hMSCs) is an attractive strategy to activate the reparative …
Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications
The recently developed CRISPR/Cas9 technology has revolutionized the genome
engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics …
engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics …
Emerging therapeutic approaches for osteogenesis imperfecta
Osteogenesis imperfecta (OI) is an incurable genetic brittle-bone disease. Although drug
therapy, surgery and physiotherapy represent current treatments for OI, the search is …
therapy, surgery and physiotherapy represent current treatments for OI, the search is …