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Rare immune diseases paving the road for genome editing-based precision medicine

Clustered regularly interspaced short palindromic repeats (CRISPR) genome editing platform heralds a new era of gene therapy. Innovative treatments for life-threatening monogenic diseases of the blood and immune system are transitioning from semi-random gene addition to precise modification of defec...

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Detalles Bibliográficos
Autores principales: Pavel-Dinu, Mara, Borna, Simon, Bacchetta, Rosa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9945114/
https://www.ncbi.nlm.nih.gov/pubmed/36846437
http://dx.doi.org/10.3389/fgeed.2023.1114996