Gene editing and CRISPR in the clinic: current and future perspectives
Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
Genome editing with the CRISPR‐Cas system: an art, ethics and global regulatory perspective
D Zhang, A Hussain, H Manghwar, K Xie… - Plant biotechnology …, 2020 - Wiley Online Library
Over the last three decades, the development of new genome editing techniques, such as
ODM, TALENs, ZFNs and the CRISPR‐Cas system, has led to significant progress in the …
ODM, TALENs, ZFNs and the CRISPR‐Cas system, has led to significant progress in the …
Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice
S Ling, S Yang, X Hu, D Yin, Y Dai, X Qian… - Nature biomedical …, 2021 - nature.com
Therapeutic genome editing requires effective and targeted delivery methods. The delivery
of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy …
of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy …
Non-integrating lentiviral vectors in clinical applications: A glance through
Lentiviral vectors (LVs) play an important role in gene therapy and have proven successful
in clinical trials. LVs are capable of integrating specific genetic materials into the target cells …
in clinical trials. LVs are capable of integrating specific genetic materials into the target cells …
NHEJ-mediated repair of CRISPR-Cas9-induced DNA breaks efficiently corrects mutations in HSPCs from patients with fanconi anemia
FJ Román-Rodríguez, L Ugalde, L Álvarez, B Díez… - Cell Stem Cell, 2019 - cell.com
Non-homologous end-joining (NHEJ) is the preferred mechanism used by hematopoietic
stem cells (HSCs) to repair double-stranded DNA breaks and is particularly increased in …
stem cells (HSCs) to repair double-stranded DNA breaks and is particularly increased in …
The old and the new: prospects for non-integrating lentiviral vector technology
L Apolonia - Viruses, 2020 - mdpi.com
Lentiviral vectors have been developed and used in multiple gene and cell therapy
applications. One of their main advantages over other vectors is the ability to integrate the …
applications. One of their main advantages over other vectors is the ability to integrate the …
Precise excision of HTLV-1 provirus with a designer-recombinase
T Rojo-Romanos, J Karpinski, S Millen, N Beschorner… - Molecular Therapy, 2023 - cell.com
The human T cell leukemia virus type 1 (HTLV-1) is a pathogenic retrovirus that persists as a
provirus in the genome of infected cells and can lead to adult T cell leukemia (ATL) …
provirus in the genome of infected cells and can lead to adult T cell leukemia (ATL) …
Rare opportunities: CRISPR/Cas-based therapy development for rare genetic diseases
Rare diseases pose a global challenge, in that their collective impact on health systems is
considerable, whereas their individually rare occurrence impedes research and …
considerable, whereas their individually rare occurrence impedes research and …
Prediction of off-target specificity and cell-specific fitness of CRISPR-Cas System using attention boosted deep learning and network-based gene feature
CRISPR-Cas is a powerful genome editing technology and has a great potential for in vivo
gene therapy. Successful translational application of CRISPR-Cas to biomedicine still faces …
gene therapy. Successful translational application of CRISPR-Cas to biomedicine still faces …
Programmable molecular scissors: applications of a new tool for genome editing in biotech
Targeted genome editing is an advanced technique that enables precise modification of the
nucleic acid sequences in a genome. Genome editing is typically performed using tools …
nucleic acid sequences in a genome. Genome editing is typically performed using tools …