Margaux Mombled
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View article: Advanced pipeline for CRISPR/Cas9 off-targets detection in Guide-seq and related integration-based assays
Advanced pipeline for CRISPR/Cas9 off-targets detection in Guide-seq and related integration-based assays Open
Background The advent of CRISPR-Cas9 genome editing has brought about a paradigm shift in molecular biology and gene therapy. However, the persistent challenge of off-target effects continues to hinder its therapeutic applications. Uninten…
View article: Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease Open
View article: Base editing of β<sup>0</sup> thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies
Base editing of β<sup>0</sup> thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies Open
Gene therapy has emerged as a promising curative treatment for β-hemoglobinopathies, the most common genetic disorders worldwide. However, current approved approaches still have some limitations in terms of safety and efficacy. Here, we us…
View article: Alarmin S100A9 restricts retroviral infection by limiting reverse transcription in human dendritic cells
Alarmin S100A9 restricts retroviral infection by limiting reverse transcription in human dendritic cells Open
View article: Impact of Aldosterone Antagonists on Sudden Cardiac Death Prevention in Heart Failure and Post-Myocardial Infarction Patients: A Systematic Review and Meta-Analysis of Randomized Controlled Trials
Impact of Aldosterone Antagonists on Sudden Cardiac Death Prevention in Heart Failure and Post-Myocardial Infarction Patients: A Systematic Review and Meta-Analysis of Randomized Controlled Trials Open
Aldosterone antagonists appear to be effective in reducing SCD and other mortality events, compared with placebo or standard medication in patients with HF and/or after a MI.