The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements

The common IVSI-110 (G>A) β-thalassemia mutation is a paradigm for intronic disease-causing mutations and their functional repair by non-homologous end joining-mediated disruption. Such mutation-specific repair by disruption of aberrant regulatory elements (DARE) is highly efficient, but to date,...

Descripción completa

Guardado en:
Detalles Bibliográficos
Publicado en:J Clin Med
Autores principales: Patsali, Petros, Mussolino, Claudio, Ladas, Petros, Floga, Argyro, Kolnagou, Annita, Christou, Soteroula, Sitarou, Maria, Antoniou, Michael N., Cathomen, Toni, Lederer, Carsten Werner, Kleanthous, Marina
Formato: Artigo
Lenguaje:Inglês
Publicado: MDPI 2019
Materias:
Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6912506/
https://ncbi.nlm.nih.gov/pubmed/31766235
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm8111959
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares