5870557
doi
10.3389/fgeed.2021.618378
oai:zenodo.org:5870557
user-upgrade-h2020-project
user-eu
Vavassori, Valentina
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy - PhD course in Molecular Medicine, Vita-Salute San Raffele University, Milan, Italy
Canarutto, Daniele
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy - PhD course in Molecular Medicine, Vita-Salute San Raffele University, Milan, Italy - Pediatric Immunohematology and Bone Marrow Transplantation Unit, Istituto di Ricovero e Cura a Carattere Scientifico Ospedale San Raffaele, Milan, Italy
Jacob, Aurelien
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy - PhD Program in Translational and Molecular Medicine (DIMET), Milano-Bicocca University, Monza, Italy
Castiello, Maria Carmina
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy - Institute of Genetic and Biomedical Research Milan Unit, National Research Council, Milan, Italy
Javed, Attya Omer
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy
Genovese, Pietro
Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA, United States - Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, United States - Harvard Stem Cell Institute, Cambridge, MA, United States - Department of Pediatrics, Harvard Medical School, Boston, MA, United States
Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation
Ferrari, Samuele
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute, Milan, Italy - PhD course in Molecular Medicine, Vita-Salute San Raffele University, Milan, Italy
info:eu-repo/semantics/openAccess
Creative Commons Attribution 4.0 International
https://creativecommons.org/licenses/by/4.0/legalcode
<p>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 nucleases, such as CRISPR/Cas9, it has been possible to expand the applications of gene therapy beyond semi-random gene addition to site-specific modification of the genome, holding the promise for safer genetic manipulation. Here we review the state of the art of <em>ex vivo</em> gene editing with programmable nucleases in human hematopoietic stem and progenitor cells (HSPCs). We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.</p>
Zenodo
2021-03-31
info:eu-repo/semantics/article
5870556
user-upgrade-h2020-project
user-eu
award_title=Unlocking Precision Gene Therapy; award_number=825825; award_identifiers_scheme=url; award_identifiers_identifier=https://cordis.europa.eu/projects/825825; funder_id=00k4n6c32; funder_name=European Commission;
1642556930.895177
1445840
md5:b2d873c9f59027b7b50adacb3d445c41
https://zenodo.org/records/5870557/files/Ferrari et al. 2021.pdf
public
Frontiers in Genome Editing
2021-03-31