Genome editing for blood disorders: state of the art and recent advances
M Romito, R Rai, AJ Thrasher… - Emerging Topics in Life …, 2019 - portlandpress.com
In recent years, tremendous advances have been made in the use of gene editing to
precisely engineer the genome. This technology relies on the activity of a wide range of …
precisely engineer the genome. This technology relies on the activity of a wide range of …
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 …
[HTML][HTML] Gene editing of hematopoietic stem cells: hopes and hurdles toward clinical translation
S Ferrari, V Vavassori, D Canarutto, A Jacob… - Frontiers in Genome …, 2021 - frontiersin.org
In the field of hematology, gene therapies based on integrating vectors have reached
outstanding results for a number of human diseases. With the advent of novel programmable …
outstanding results for a number of human diseases. With the advent of novel programmable …
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] CRISPR to fix bad blood: a new tool in basic and clinical hematology
E González-Romero, C Martínez-Valiente… - …, 2019 - ncbi.nlm.nih.gov
Advances in genome engineering in the last decade, particularly in the development of
programmable nucleases, have made it possible to edit the genomes of most cell types …
programmable nucleases, have made it possible to edit the genomes of most cell types …
Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective
M Amendola, A Bedel, A Buj-Bello, M Carrara… - Human gene …, 2021 - liebertpub.com
Recent advances in genome editing tools, especially novel developments in the clustered
regularly interspaced short palindromic repeats associated to Cas9 nucleases …
regularly interspaced short palindromic repeats associated to Cas9 nucleases …
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] Gene editing for inherited red blood cell diseases
O Quintana-Bustamante, S Fañanas-Baquero… - Frontiers in …, 2022 - frontiersin.org
Today gene therapy is a real therapeutic option to address inherited hematological diseases
that could be beneficial for thousands of patients worldwide. Currently, gene therapy is used …
that could be beneficial for thousands of patients worldwide. Currently, gene therapy is used …
Editing human hematopoietic stem cells: advances and challenges
Genome editing of hematopoietic stem and progenitor cells is being developed for the
treatment of several inherited disorders of the hematopoietic system. The adaptation of …
treatment of several inherited disorders of the hematopoietic system. The adaptation of …
[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 …