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Therapy: Gene Therapy

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Total 60 results found since Jan 2013.

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

Heterogeneity of the long-term economic burden of severe sickle cell disease: a 5-year longitudinal analysis
CONCLUSION: Results indicate high clinical need and economic burden among patients with severe presentation of SCD. These findings not only highlight the need for improved therapeutic options to limit or prevent disease progression, but also starts to provide insight on lifetime costs of SCD that will be needed in the evaluation of emerging curative intent therapies.PMID:36222016 | DOI:10.1080/13696998.2022.2133824
Source: Journal of Medical Economics - October 12, 2022 Category: Health Management Authors: Meghan Gallagher Anjulika Chawla Brenna L Brady Sherif M Badawy Source Type: research

Genes, Vol. 13, Pages 144: Influence of Haptoglobin Polymorphism on Stroke in Sickle Cell Disease Patients
vain Doré This review outlines the current clinical research investigating how the haptoglobin (Hp) genetic polymorphism and stroke occurrence are implicated in sickle cell disease (SCD) pathophysiology. Hp is a blood serum glycoprotein responsible for binding and removing toxic free hemoglobin from the vasculature. The role of Hp in patients with SCD is critical in combating blood toxicity, inflammation, oxidative stress, and even stroke. Ischemic stroke occurs when a blocked vessel decreases oxygen delivery in the blood to cerebral tissue and is commonly associated with SCD. Due to the malformed red blood cells of s...
Source: Genes - January 14, 2022 Category: Genetics & Stem Cells Authors: Olivia Edwards Alicia Burris Josh Lua Diana J. Wilkie Miriam O. Ezenwa Sylvain Dor é Tags: Review Source Type: research

Genes, Vol. 13, Pages 48: Ischemic Stroke Genetics: What Is New and How to Apply It in Clinical Practice?
Jatužis The etiology of ischemic stroke is multifactorial. Although receiving less emphasis, genetic causes make a significant contribution to ischemic stroke genesis, especially in early-onset stroke. Several stroke classification systems based on genetic information corresponding to various stroke phenotypes were proposed. Twin and family history studies, as well as candidate gene approach, are common methods to discover genetic causes of stroke, however, both have their own limitations. Genome-wide association studies and next generation sequencing are more efficient, promising and increasingly used for daily diagn...
Source: Genes - December 24, 2021 Category: Genetics & Stem Cells Authors: Aleksandra Ekkert Aleksandra Šliachtenko Julija Grigait ė Birut ė Burnytė Algirdas Utkus Dalius Jatu žis Tags: Review Source Type: research

Mesenchymal Stromal Cells (MSCs): a promising tool for cell-based angiogenic therapy
Curr Gene Ther. 2021 Sep 16. doi: 10.2174/1566523221666210917114353. Online ahead of print.ABSTRACTThe Mesenchymal stromal cells (MSCs) are a diverse subset of adult multipotent precursors, known for their potential therapeutic properties in regenerative medicine mainly sustained by paracrine effects, through secretion of a variety of biologically active molecules. MSC secretome includes a wide range of soluble protein factors, composed of growth factors and cytokines, and vesicular components, which transfer proteins and genetic material modulating the host microenvironment. In particular, MSC-derived secretome mediates t...
Source: Current Gene Therapy - September 17, 2021 Category: Genetics & Stem Cells Authors: Cristiana Ulpiano Cl áudia L da Silva Gabriel A Monteiro Source Type: research