Application of genome editing technologies to the study and treatment of hematological disease

A Pellagatti, H Dolatshad, BH Yip, S Valletta… - Advances in biological …, 2016 - Elsevier
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 …

Gene editing and its application for hematological diseases

MJ Osborn, JJ Belanto, J Tolar, DF Voytas - International journal of …, 2016 - Springer
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 …

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 …

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 …

Genome editing for human gene therapy

TB Meissner, PK Mandal, LMR Ferreira, DJ Rossi… - Methods in …, 2014 - Elsevier
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 …

Tools for efficient genome editing; ZFN, TALEN, and CRISPR

Y Shamshirgaran, J Liu, H Sumer, PJ Verma… - Applications of genome …, 2022 - Springer
The last two decades have marked significant advancement in the genome editing field.
Three generations of programmable nucleases (ZFNs, TALENs, and CRISPR-Cas system) …

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 …

Targeted genome editing tools for disease modeling and gene therapy

M Cai, Y Yang - Current gene therapy, 2014 - ingentaconnect.com
The development of custom-designed nucleases (CDNs), including zinc finger nucleases
(ZFNs) and transcription activator-like effector nucleases (TALENs), has made it possible to …

Programmable molecular scissors: applications of a new tool for genome editing in biotech

SK Saha, FK Saikot, MS Rahman, MAHM Jamal… - … Therapy-Nucleic Acids, 2019 - cell.com
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 …

Gene therapy and gene editing in healthcare

S Dwivedi, P Purohit, A Vasudeva, M Kumar… - Biotechnology in …, 2022 - Elsevier
Gene therapy refers to the addition of new genes to human cells, while genome editing
involves the precise manipulation of the sequence of the human genome for disease …