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Gene therapy of adeno-associated virus (AAV) vectors in preclinical models of ischemic stroke
CNS Neurosci Ther. 2023 Aug 8. doi: 10.1111/cns.14392. Online ahead of print.ABSTRACTStroke has been associated with devastating clinical outcomes, with current treatment strategies proving largely ineffective. Therefore, there is a need to explore alternative treatment options for addressing post-stroke functional deficits. Gene therapy utilizing adeno-associated viruses (AAVs) as a critical gene vector delivering genes to the central nervous system (CNS) gene delivery has emerged as a promising approach for treating various CNS diseases. This review aims to provide an overview of the biological characteristics of AAV vec...
Source: CNS Neuroscience and Therapeutics - August 8, 2023 Category: Neuroscience Authors: Jing Wang Mengna Zhu Jingyi Sun Lina Feng Mingfeng Yang Baoliang Sun Leilei Mao Source Type: research

Lipid Lowering Therapy: An Era Beyond Statins
Curr Probl Cardiol. 2022 Jul 30:101342. doi: 10.1016/j.cpcardiol.2022.101342. Online ahead of print.ABSTRACTDyslipidemia, specifically elevated LDL cholesterol levels, causes atherosclerotic cardiovascular disease (ASCVD) and increases the risk of myocardial infarction and stroke. Statins, a class of drugs that exert their effects by inhibiting HMG-CoA reductase, a key enzyme in the synthesis of cholesterol, have been the mainstay of therapy for the primary prevention of cardiovascular disease and lipids reduction. Statins are associated with side effects, most commonly myopathy and myalgias, despite their proven efficacy....
Source: Atherosclerosis - August 2, 2022 Category: Cardiology Authors: Toufik Abdul-Rahman Syed Muhammad Awais Bukhari Emiliano Cantu Herrera Wireko Andrew Awuah Jannel Lawrence Heloisa de Andrade Neal Patel Rohan Shah Raheel Shaikh Camilo Andr és Avendaño Capriles Sebahat Ulusan Shahzaib Ahmad Anna Corriero Adriana C Mare Source Type: research

Po-626-03 preclinical safety of adkcnh2-g628s for post-op af
Post-operative atrial fibrillation (POAF) occurs in 30% of cardiac surgeries, increasing risk of stroke, MI, and death. We propose gene therapy with an adenovirus encoding the G628S mutation of KCNH2 (AdKCNH2-G628S) to prevent POAF. We have reported preclinical efficacy in pigs.
Source: Heart Rhythm - April 29, 2022 Category: Cardiology Authors: J. Kevin Donahue, David D. McManus Source Type: research

Peripherally delivered Adeno-associated viral vectors for spinal cord injury repair
In conclusion, in the future, minimally invasive administration of AAVs may improve recovery after SCI with minimal side effects.PMID:34896114 | DOI:10.1016/j.expneurol.2021.113945
Source: Experimental Neurology - December 13, 2021 Category: Neurology Authors: Jared D Sydney-Smith Aline B Spejo Philippa M Warren Lawrence D F Moon Source Type: research

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

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Conclusions: Collectively, these data provide strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease. We anticipate that a single dose IV procedure will pose minimal burden to Fabry patients and will be a viable alternative to biweekly enzyme infusions, potentially reducing treatment-related morbidity whislt improving patient quality of life and potentially providing them with a functional long-term cure.DisclosuresKia: Freeline: Employment, Equity Ownership. McIntosh: Freeline: Consultancy. Hosseini: Freeline: Employment, Equit...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., Mills, K., Corbau, R., Nathwani, A. C. Tags: 801. Gene Therapy and Transfer: Poster I Source Type: research

Neuroscience is the Next Oncology
by Michael D. Ehlers, MD, PhD Dr. Ehlers is with Biogen in Cambridge, Massachusetts. Innov Clin Neurosci. 2018;15(3–4):15–16 Funding: No funding was received for the preparation of this article. Disclosures: Dr. Ehlers is an employee and shareholder at Biogen Inc. in Cambridge, Massachusetts. Prominent and expensive failures in Alzheimer’s disease therapies have led to a contagious belief system in some parts of the biopharma industry that neuroscience is just too hard, too risky, and too uncertain. But, might this belief system itself be a residual bias of the past? Close inspection reveals all the signs of a coming...
Source: Innovations in Clinical Neuroscience - April 1, 2018 Category: Neuroscience Authors: ICNS Online Editor Tags: Commentary Current Issue Source Type: research

A Review of Mathematical Models for Muscular Dystrophy: A Systems Biology Approach
Discussion With new developments in computational power and data availability, a growing amount of research is using a systems biology approach to understand pathogenesis and progression of disease. Effective and integrated in vitro and in silico models could inform biological phenomena, even without the need of a living subject. For instance, over the last few decades, collagen hydrogel with muscle derived cells (CHMDCs) have promised to revolutionize in vitro experiments and tissue engineering. For CHMDCs to reach the envisioned use, verification by use of mathematical simulations are needed. Recently while examining sha...
Source: PLOS Currents Muscular Dystrophy - February 16, 2018 Category: Neurology Authors: Matthew Houston Source Type: research

Effect of combined VEGF165/SDF-1 gene therapy on vascular remodeling and blood perfusion in cerebral ischemia.
CONCLUSIONS Combined VEGF165 and SDF-1 gene therapy represents a potential strategy for improving vascular remodeling and recovery of neural function after cerebral infarction. PMID: 27982773 [PubMed - as supplied by publisher]
Source: Journal of Neurosurgery - December 15, 2016 Category: Neurosurgery Authors: Hu GJ, Feng YG, Lu WP, Li HT, Xie HW, Li SF Tags: J Neurosurg Source Type: research

Comparison of vascular growth factors in the murine brain reveals placenta growth factor as prime candidate for CNS revascularization
Vascular bypass procedures in the central nervous system (CNS) remain technically challenging, hindered by complications and often failing to prevent adverse outcome such as stroke. Thus, there is an unmet clinical need for a safe and effective CNS revascularization. Vascular endothelial growth factors (VEGFs) are promising candidates for revascularization; however, their effects appear to be tissue-specific and their potential in the CNS has not been fully explored. To test growth factors for angiogenesis in the CNS, we characterized the effects of endothelium-specific growth factors on the brain vasculature and parenchym...
Source: Blood - August 1, 2013 Category: Hematology Authors: Gaal, E. I., Tammela, T., Anisimov, A., Marbacher, S., Honkanen, P., Zarkada, G., Leppanen, V.-M., Tatlisumak, T., Hernesniemi, J., Niemela, M., Alitalo, K. Tags: Vascular Biology, Gene Therapy Source Type: research