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Therapeutic potential of AAV9-S15D-RLC gene delivery in humanized MYL2 mouse model of HCM
This study is focused on aspartic acid-to-valine (D166V) mutation in the myosin regulatory light chain, RLC (MYL2 gene), associated with a malignant form of HCM. Since myosin RLC phosphorylation is critical for normal cardiac function, we aimed to exploit this post-translational modification via phosphomimetic-RLC gene therapy. We hypothesized that mimicking/modulating cardiac RLC phosphorylation in non-phosphorylatable D166V myocardium would improve heart function of HCM-D166V mice. Adeno-associated virus, serotype-9 (AAV9) was used to deliver phosphomimetic human RLC variant with serine-to-aspartic acid substitution at S...
Source: Journal of Molecular Medicine - May 16, 2019 Category: Molecular Biology Source Type: research

Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases
This article mainly focuses on the delivering modes of genetic materials in the CNS, which includes viral and non-viral vectors and their application in gene therapy. Despite the many clinical trials conducted so far, data have shown disappointing outcomes. The efforts done to improve outcomes, efficacy, and safety in the identification of targets in various neurological disorders are also discussed here. Adapting gene therapy as a new therapeutic approach for treating neurological disorders seems to be promising, with early detection and delivery of therapy before the neuron is lost, helping a lot the development of new t...
Source: Molecular Medicine - October 16, 2021 Category: Molecular Biology Authors: Della Grace Thomas Parambi Khalid Saad Alharbi Rajesh Kumar Seetha Harilal Gaber El-Saber Batiha Nat ália Cruz-Martins Omnia Magdy Arafa Musa Dibya Sundar Panda Bijo Mathew Source Type: research