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Therapy: Gene Therapy
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Total 5568 results found since Jan 2013.
Successful Transduction of Target Gene Mediated by Adeno-Associated Virus 2 into Lens Epithelial Cells in Rats
In this study, we compared the efficiency of intravitreal injection of six AAV serotypes (AAV2, AAV5, AAV6, AAV8, AAV9, and AAVDJ) to transduce lens and retina in rats, The expression and localization of the reporter gene ZsGreen in the lens and retina were examined using immunofluorescence staining, and the relative expression of ZsGreen mRNA was detected using RT-qPCR. Our results demonstrated that AAV2 had the highest efficiency in transducing LECs. All six AAV serotypes could transduce the retina. To validate this observation, we further constructed an AAV2 vector with exogenous gene senescence marker protein 30 (SMP30...
Source: Journal of Virological Methods - August 17, 2023 Category: Virology Authors: Yongshun Liang Tian Lan Qingqiao Gan Hao Liang Source Type: research
Expression of two major isoforms of MYO7A in the retina: Considerations for gene therapy of Usher syndrome type 1B
Vision Res. 2023 Aug 14;212:108311. doi: 10.1016/j.visres.2023.108311. Online ahead of print.ABSTRACTUsher syndrome type 1B (USH1B) is a deaf-blindness disorder, caused by mutations in the MYO7A gene, which encodes the heavy chain of an unconventional actin-based motor protein. Here, we examined the two retinal isoforms of MYO7A, IF1 and IF2. We compared 3D models of the two isoforms and noted that the 38-amino acid region that is present in IF1 but absent from IF2 affects the C lobe of the FERM1 domain and the opening of a cleft in this potentially important protein binding domain. Expression of each of the two isoforms o...
Source: Vision Research - August 16, 2023 Category: Opthalmology Authors: W Blake Gilmore Nan W Hultgren Abhishek Chadha Sonia B Barocio Joyce Zhang Oksana Kutsyr Miguel Flores-Bellver M Valeria Canto-Soler David S Williams Source Type: research
An update on multiplexed mass spectrometry-based lysosomal storage disease diagnosis
Mass Spectrom Rev. 2023 Aug 16. doi: 10.1002/mas.21864. Online ahead of print.ABSTRACTLysosomal storage disorders (LSDs) are a type of inherited metabolic disorders in which biomolecules, accumulate as a specific substrate in lysosomes due to specific individual enzyme deficiencies. Despite the fact that LSDs are incurable, various approaches, including enzyme replacement therapy, hematopoietic stem cell transplantation, or gene therapy are now available. Therefore, a timely diagnosis is a critical initial step in patient treatment. The-state-of-the-art in LSD diagnostic uses, in the first stage, enzymatic activity determi...
Source: Mass Spectrometry Reviews - August 16, 2023 Category: Molecular Biology Authors: Laura Darie-Ion Br înduşa Alina Petre Source Type: research
Expression of two major isoforms of MYO7A in the retina: Considerations for gene therapy of Usher syndrome type 1B
Vision Res. 2023 Aug 14;212:108311. doi: 10.1016/j.visres.2023.108311. Online ahead of print.ABSTRACTUsher syndrome type 1B (USH1B) is a deaf-blindness disorder, caused by mutations in the MYO7A gene, which encodes the heavy chain of an unconventional actin-based motor protein. Here, we examined the two retinal isoforms of MYO7A, IF1 and IF2. We compared 3D models of the two isoforms and noted that the 38-amino acid region that is present in IF1 but absent from IF2 affects the C lobe of the FERM1 domain and the opening of a cleft in this potentially important protein binding domain. Expression of each of the two isoforms o...
Source: Vision Research - August 16, 2023 Category: Opthalmology Authors: W Blake Gilmore Nan W Hultgren Abhishek Chadha Sonia B Barocio Joyce Zhang Oksana Kutsyr Miguel Flores-Bellver M Valeria Canto-Soler David S Williams Source Type: research
An update on multiplexed mass spectrometry-based lysosomal storage disease diagnosis
Mass Spectrom Rev. 2023 Aug 16. doi: 10.1002/mas.21864. Online ahead of print.ABSTRACTLysosomal storage disorders (LSDs) are a type of inherited metabolic disorders in which biomolecules, accumulate as a specific substrate in lysosomes due to specific individual enzyme deficiencies. Despite the fact that LSDs are incurable, various approaches, including enzyme replacement therapy, hematopoietic stem cell transplantation, or gene therapy are now available. Therefore, a timely diagnosis is a critical initial step in patient treatment. The-state-of-the-art in LSD diagnostic uses, in the first stage, enzymatic activity determi...
Source: Mass Spectrometry Reviews - August 16, 2023 Category: Molecular Biology Authors: Laura Darie-Ion Br înduşa Alina Petre Source Type: research
Recent advances in drug delivery approaches for rheumatoid arthritis
Curr Med Chem. 2023 Aug 15. doi: 10.2174/0929867331666230815112818. Online ahead of print.ABSTRACTMorbidity, disability, and healthcare expenses associated with rheumatoid arthritis (RA) impose a considerable health and economical burden on both patients and healthcare systems. This review aimed to examine the pathophysiological aspects of RA that may help design different types of drugs and drug delivery systems. These include monoclonal antibodies, immunoglobulins, tiny chemicals, and transgenes for gene therapy. These novel nanocarrier-based therapies target the underlying biological processes involved in RA while minim...
Source: Current Medicinal Chemistry - August 15, 2023 Category: Chemistry Authors: Abhishek Verma Preeti Patel Waleed H Almalki Amirhossein Sahebkar Balak Das Kurmi Prashant Kesharwani Source Type: research
Fight Aging! Newsletter, August 14th 2023
This study demonstrates just how vital the thymus is to maintaining adult health."
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Does Amyloid-β Aggregation Cause Broad Disruption of Proteostasis?
https://www.fightaging.org/archives/2023/08/does-amyloid-%ce%b2-aggregation-cause-broad-disruption-of-proteostasis/
Researchers here speculate on the ability of insoluble amyloid-β aggregates to be broadly disruptive of the solubility of many other proteins, and thus disruptive to cell and tissue function. Is this important in aging? The evidence here shows the existence of the mechanism in a lower species, but that doesn't n...
Source: Fight Aging! - August 13, 2023 Category: Research Authors: Reason Tags: Newsletters Source Type: blogs
FAM64A aggravates proliferation, invasion, lipid droplet formation, and chemoresistance in gastric cancer: A biomarker for aggressiveness and a gene therapy target
Drug Dev Res. 2023 Aug 11. doi: 10.1002/ddr.22105. Online ahead of print.ABSTRACTFAM64A is a mitogen-induced regulator of the metaphase and anaphase transition. Here, we found that FAM64A messenger RNA (mRNA) and protein expression levels were higher in gastric cancer tissue than in normal mucosa (p < .05). FAM64A methylation was negatively correlated with FAM64A mRNA expression (p < .05). The differentially expressed genes of FAM64A were mainly involved in digestion, potassium transporting or exchanging ATPase, contractile fibers, endopeptidase, and pancreatic secretion (p < .05). The FAM64A-related genes were pr...
Source: Cell Research - August 12, 2023 Category: Cytology Authors: Wen-Jing Yun Li Zhang Ning Yang Zheng-Guo Cui Hua-Mao Jiang Min-Wen Ha Da-Yong Yu Ming-Zhen Zhao Hua-Chuan Zheng Source Type: research
Effect of pharmacological heart failure drugs and gene therapy on Danon's cardiomyopathy
Biochem Pharmacol. 2023 Aug 10:115735. doi: 10.1016/j.bcp.2023.115735. Online ahead of print.ABSTRACTDanon disease is an X-linked genetic disease resulting from LAMP2 mutations leading to defective lysosomal function. Heart failure is the main causes of morbidity and mortality. Mice with an LAMP2-exon-6-deletion (L2Δ6), develop cardiac hypertrophy followed by dilated cardiomyopathy, in association with accumulation of autophagosomes, fibrosis and oxidative stress. We investigated the effect of drugs used to treat heart failure and of LAMP2 gene therapy on the phenotype, molecular markers and ROS in LAMP2 cardiomyopathy. L...
Source: Biochemical Pharmacology - August 12, 2023 Category: Drugs & Pharmacology Authors: Dor Yadin Tali Guetta Zachary Petrover Ronny Alcalai Jon Seidman Christine E Seidman Efrat Ofek Ran Kornowski Edith Hochhauser Michael Arad Source Type: research
Pioneering therapies for post-infarction angiogenesis: Insight into molecular mechanisms and preclinical studies
Biomed Pharmacother. 2023 Aug 10;166:115306. doi: 10.1016/j.biopha.2023.115306. Online ahead of print.ABSTRACTAcute myocardial infarction (MI), despite significant progress in its treatment, remains a leading cause of chronic heart failure and cardiovascular events such as cardiac arrest. Promoting angiogenesis in the myocardial tissue after MI to restore blood flow in the ischemic and hypoxic tissue is considered an effective treatment strategy. The repair of the myocardial tissue post-MI involves a robust angiogenic response, with mechanisms involved including endothelial cell proliferation and migration, capillary growt...
Source: Biomedicine and pharmacotherapy = Biomedecine and pharmacotherapie - August 12, 2023 Category: Drugs & Pharmacology Authors: Cong Chen Jie Wang Chao Liu Jun Hu Lanchun Liu Source Type: research
