[HTML][HTML] Gene editing and its application for hematological diseases
The use of precise, rationally designed gene-editing nucleases allows for targeted genome
and transcriptome modification, and at present, four major classes of nucleases are being …
and transcriptome modification, and at present, four major classes of nucleases are being …
Application of genome editing technologies to the study and treatment of hematological disease
Genome editing technologies have advanced significantly over the past few years, providing
a fast and effective tool to precisely manipulate the genome at specific locations. The three …
a fast and effective tool to precisely manipulate the genome at specific locations. The three …
Genome and epigenome editing to treat disorders of the hematopoietic system
C Mussolino, J Alzubi, V Pennucci, G Turchiano… - Human gene …, 2017 - liebertpub.com
The possibility of editing complex genomes in a targeted fashion has revolutionized basic
research as well as biomedical and biotechnological applications in the last 5 years. The …
research as well as biomedical and biotechnological applications in the last 5 years. The …
[HTML][HTML] Genome-editing technologies for gene and cell therapy
ML Maeder, CA Gersbach - Molecular therapy, 2016 - cell.com
Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
However, the recent advent of genome-editing technologies has enabled a new paradigm in …
Gene editing of human hematopoietic stem and progenitor cells: promise and potential hurdles
Hematopoietic stem and progenitor cells (HSPCs) have great therapeutic potential because
of their ability to both self-renew and differentiate. It has been proposed that, given their …
of their ability to both self-renew and differentiate. It has been proposed that, given their …
[HTML][HTML] In vivo hematopoietic stem cell genome editing: perspectives and limitations
The tremendous evolution of genome-editing tools in the last two decades has provided
innovative and effective approaches for gene therapy of congenital and acquired diseases …
innovative and effective approaches for gene therapy of congenital and acquired diseases …
Genome editing for human gene therapy
The rapid advancement of genome-editing techniques holds much promise for the field of
human gene therapy. From bacteria to model organisms and human cells, genome editing …
human gene therapy. From bacteria to model organisms and human cells, genome editing …
[HTML][HTML] Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …
technologies has opened up the possibility of directly targeting and modifying genomic …
CRISPR/Cas9 system: A promising technology for the treatment of inherited and neoplastic hematological diseases
JS Antony, AKMA Haque… - Advances in Cell …, 2018 - Wiley Online Library
The ongoing advent of genome editing with programmable nucleases, including zinc‐finger
nuclease (ZFN), TAL effector nuclease (TALEN), and Clustered Regularly Interspaced Short …
nuclease (ZFN), TAL effector nuclease (TALEN), and Clustered Regularly Interspaced Short …
Therapeutic genome editing with engineered nucleases
SA Haas, V Dettmer, T Cathomen - Hämostaseologie, 2017 - thieme-connect.com
Targeted genome editing with designer nucleases, such as zinc finger nucleases, TALE
nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic …
nucleases, and CRISPR-Cas nucleases, has heralded a new era in gene therapy. Genetic …