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Therapy: Gene Therapy

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Total 5568 results found since Jan 2013.

Clinical trials in Charcot-Marie-Tooth disorders: a retrospective and preclinical assessment
ConclusionCurrent CMT trials are exploring procedural and molecular therapeutic options with substantial participation of the pharmaceutical industry worldwide. Emerging drug therapies directed at molecular pathogenesis are being advanced in human clinical trials; however, the majority remain within animal investigations.
Source: Frontiers in Neurology - September 22, 2023 Category: Neurology Source Type: research

Many Researchers and Companies Will Aim to Produce Small Molecule Reprogramming Therapies
The typical path for any program in biomedical research and development is to first demonstrate interesting results in animal studies using forms of genetic engineering or gene therapy, and then find small molecules that adjust the same mechanism. Small molecules are never as good as genetic manipulations, the size of the effect is always smaller, usually much smaller, and there are inevitably side-effects. Small molecule development is much easier to conduct, however, more familiar to investors and regulators and program managers, a well-trodden path. Thus while the future of medicine is gene therapy, in search of large e...
Source: Fight Aging! - September 21, 2023 Category: Research Authors: Reason Tags: Daily News Source Type: blogs

TRPM8 as a Potential Biomarker and Therapeutic Target for Gastric Cancer Identified by a Combination of Text Mining and RNA Sequencing
CONCLUSION: This study provides insights into the underlying role of TRPM8 in cell proliferation. In addition, TRPM8 may be used as a potential therapeutic target for patients with gastric cancer.PMID:37728085 | DOI:10.2174/1566523223666230529142423
Source: Current Gene Therapy - September 20, 2023 Category: Genetics & Stem Cells Authors: Na Kong Wendong Li Jun Zhang Xin Wang Lin Hu Qiqi Xu Source Type: research

Exosomal miRNAs as Next-generation Therapy Vehicles in Breast Cancer
Curr Gene Ther. 2023;23(5):330-342. doi: 10.2174/1566523223666230215103524.ABSTRACTThe second most pervasive cancer affecting the survival of women across the world is breast cancer. One of the biggest challenges in breast cancer treatment is the chemoresistance of cancer cells to various medications after some time. Therefore, highly specific blood-based biomarkers are required for early breast cancer diagnosis to overcome chemoresistance and improve patient survival. These days, exosomal miRNAs have attracted much attention as early diagnostic blood-based biomarkers because of their high stability, secretion from maligna...
Source: Current Gene Therapy - September 20, 2023 Category: Genetics & Stem Cells Authors: Priyanka Thakur Harshita Dahiya Ankur Kaushal Vijai Kumar Gupta Adesh K Saini Reena V Saini Source Type: research

MicroRNA Theranostics in Cancer
Curr Gene Ther. 2023;23(5):329. doi: 10.2174/156652322305230808102847.NO ABSTRACTPMID:37728083 | DOI:10.2174/156652322305230808102847
Source: Current Gene Therapy - September 20, 2023 Category: Genetics & Stem Cells Authors: Sujay Paul Source Type: research

Rational design and experimental evaluation of peptide ligands for the purification of adeno-associated viruses via affinity chromatography
This article is protected by copyright. All rights reserved.PMID:37728197 | DOI:10.1002/biot.202300230
Source: Biotechnology Journal - September 20, 2023 Category: Biotechnology Authors: Shriarjun Shastry Wenning Chu Eduardo Barbieri Paul Greback-Clarke William K Smith Christopher Cummings Arianna Minzoni Jennifer Pancorbo Gary Gilleskie Kimberly Ritola Michael A Daniele Thomas F Johnson Stefano Menegatti Source Type: research

TRPM8 as a Potential Biomarker and Therapeutic Target for Gastric Cancer Identified by a Combination of Text Mining and RNA Sequencing
CONCLUSION: This study provides insights into the underlying role of TRPM8 in cell proliferation. In addition, TRPM8 may be used as a potential therapeutic target for patients with gastric cancer.PMID:37728085 | DOI:10.2174/1566523223666230529142423
Source: Current Gene Therapy - September 20, 2023 Category: Genetics & Stem Cells Authors: Na Kong Wendong Li Jun Zhang Xin Wang Lin Hu Qiqi Xu Source Type: research

Exosomal miRNAs as Next-generation Therapy Vehicles in Breast Cancer
Curr Gene Ther. 2023;23(5):330-342. doi: 10.2174/1566523223666230215103524.ABSTRACTThe second most pervasive cancer affecting the survival of women across the world is breast cancer. One of the biggest challenges in breast cancer treatment is the chemoresistance of cancer cells to various medications after some time. Therefore, highly specific blood-based biomarkers are required for early breast cancer diagnosis to overcome chemoresistance and improve patient survival. These days, exosomal miRNAs have attracted much attention as early diagnostic blood-based biomarkers because of their high stability, secretion from maligna...
Source: Current Gene Therapy - September 20, 2023 Category: Genetics & Stem Cells Authors: Priyanka Thakur Harshita Dahiya Ankur Kaushal Vijai Kumar Gupta Adesh K Saini Reena V Saini Source Type: research

MicroRNA Theranostics in Cancer
Curr Gene Ther. 2023;23(5):329. doi: 10.2174/156652322305230808102847.NO ABSTRACTPMID:37728083 | DOI:10.2174/156652322305230808102847
Source: Current Gene Therapy - September 20, 2023 Category: Genetics & Stem Cells Authors: Sujay Paul Source Type: research

Pathophysiological Aspects and Therapeutic Armamentarium of Alzheimer's Disease: Recent Trends and Future Development
Cell Mol Neurobiol. 2023 Sep 19. doi: 10.1007/s10571-023-01408-7. Online ahead of print.ABSTRACTAlzheimer's disease (AD) is the primary cause of dementia and is characterized by the death of brain cells due to the accumulation of insoluble amyloid plaques, hyperphosphorylation of tau protein, and the formation of neurofibrillary tangles within the cells. AD is also associated with other pathologies such as neuroinflammation, dysfunction of synaptic connections and circuits, disorders in mitochondrial function and energy production, epigenetic changes, and abnormalities in the vascular system. Despite extensive research con...
Source: Cellular and Molecular Neurobiology - September 19, 2023 Category: Cytology Authors: Bhavarth P Dave Yesha B Shah Kunal G Maheshwari Kaif A Mansuri Bhadrawati S Prajapati Humzah I Postwala Mehul R Chorawala Source Type: research

Unleashing the potential of CRISPR multiplexing: Harnessing Cas12 and Cas13 for precise gene modulation in eye diseases
Vision Res. 2023 Sep 16;213:108317. doi: 10.1016/j.visres.2023.108317. Online ahead of print.ABSTRACTGene therapy is a flourishing field with the potential to revolutionize the treatment of genetic diseases. The emergence of CRISPR-Cas9 has significantly advanced targeted and efficient genome editing. Although CRISPR-Cas9 has demonstrated promising potential applications in various genetic disorders, it faces limitations in simultaneously targeting multiple genes. Novel CRISPR systems, such as Cas12 and Cas13, have been developed to overcome these challenges, enabling multiplexing and providing unique advantages. Cas13, in...
Source: Cell Research - September 18, 2023 Category: Cytology Authors: Fabio Bigini Soo Hyeon Lee Young Joo Sun Yang Sun Vinit B Mahajan Source Type: research

Autoimmune responses and therapeutic interventions for systemic lupus erythematosus: a comprehensive review
Endocr Metab Immune Disord Drug Targets. 2023 Sep 15. doi: 10.2174/1871530323666230915112642. Online ahead of print.ABSTRACTSystemic Lupus Erythematosus (SLE) or Lupus is a multifactorial autoimmune disease of multiorgan malfunctioning of extremely heterogeneous and unclear etiology that affects multiple organs and physiological systems. Some racial groups and women of childbearing age are more susceptible to SLE pathogenesis. Impressive progress has been made towards a better understanding of different immune components contributing to SLE pathogenesis. Recent investigations have uncovered the detailed mechanisms of infla...
Source: Endocrine, Metabolic and Immune Disorders Drug Targets - September 18, 2023 Category: Endocrinology Authors: Surya Prakash Pandey Rakesh Bhaskar Sung Soon Han Kannan Badri Narayanan Source Type: research