Gene therapy and editing: Novel potential treatments for neuronal channelopathies

Potential Application of Novel DNA Editing Techniques in Translational Neuroscience

No abstract is provided.

Epileptic Channelopathies and Dysfunctional Excitability - From Gene Mutations to Novel Treatments

Therapeutic approaches in patients with β-thalassemia

Beta-thalassemia (β-thal) is a congenital hemoglobinopathy explained by a decreased level (β+) or absence (βο) of β-globin gene expression. Microcytic hypochromic anemia and various clinical symptoms comprising severe anemia to clinically nonsymptomatic features. Treatment with an ordered blood transfusion and iron chelator agents can decrease transfusion iron overload that causes normal matura...

Editing of the MALAT1 Gene in MDA-MB-361 Breast Cancer Cell Line using the Novel CRISPR Method

Introduction: Long non-coding RNAs play an important role in regulating gene expression, RNA processing, histone modification, and rearrangement of chromatin genes. These molecules can also be involved in many biological processes, such as organogenesis, cell differentiation, development, genome imprinting, quantitative compensation, and tumorigenesis. High expression of MALAT1 (a type of lncRN...

Genetic and Functional S of Severe V Arrhythmias

..... ..................................................................................................................................................................... 9 INTRODUCTION .................................................................................................................................................................. 11 REVIEW OF THE LITERATURE ......................