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,...

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Vydáno v:J Clin Med
Hlavní autoři: Patsali, Petros, Mussolino, Claudio, Ladas, Petros, Floga, Argyro, Kolnagou, Annita, Christou, Soteroula, Sitarou, Maria, Antoniou, Michael N., Cathomen, Toni, Lederer, Carsten Werner, Kleanthous, Marina
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2019
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Article
On-line přístup: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
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