Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β(0)39-thalassemia patients

Gene editing by the CRISPR-Cas9 nuclease system technology can be considered among the most promising strategies to correct hereditary mutations in a variety of monogenic diseases. In this paper, we present for the first time the correction, by CRISPR-Cas9 gene editing, of the β(0)39-thalassemia mut...

Descrizione completa

Salvato in:
Dettagli Bibliografici
Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Cosenza, Lucia Carmela, Gasparello, Jessica, Romanini, Nicola, Zurlo, Matteo, Zuccato, Cristina, Gambari, Roberto, Finotti, Alessia
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2021
Soggetti:
Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8091488/
https://ncbi.nlm.nih.gov/pubmed/33997100
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.03.025
Tags: Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne! !

Documenti analoghi