AAV Vectors in Cancer Therapy: A Review of Applications and Strategies
1. Introduction Cancer continues to be a major health concern despite progress in traditional treatments like surgery, chemotherapy, and radiotherapy. Gene therapy provides an innovative approach by introducing therapeutic genes to cancer cells, enabling targeted...
Analysis of Adeno-Associated Virus Tropism in Mice: A Comprehensive Survey of AAV Serotypes
Adeno-associated viruses (AAVs) have become essential tools in gene therapy due to their ability to deliver genetic material into targeted tissues efficiently. Recent studies have focused on understanding the tropism—or tissue-targeting abilities—of various AAV...
Advances in AAV-SB Transposon Hybrid Systems for Liver-Targeted Gene Therapies
Liver-targeted gene therapies are at the forefront of treating genetic diseases, particularly with the development of adeno-associated virus (AAV) and Sleeping Beauty (SB) transposon systems. These technologies offer a range of benefits for both pediatric and adult...
Novel Approach in T Cell Engineering: Lipid Nanoparticles Enable Advanced Genome Editing for Cancer Therapies
Revolutionizing CAR T Cell Therapy with Lipid Nanoparticles Chimeric antigen receptor (CAR) T cell therapy has transformed cancer treatment by turning a patient’s own T cells into powerful cancer-fighting agents. However, as the technology advances, there is an...
New Lipid Nanoparticles Deliver CRISPR-Cas9 to Knock Down Angptl3 in Mice
A team of researchers has developed a new way to deliver CRISPR-Cas9 genome editing tools directly to the liver, targeting a gene called Angptl3 that’s linked to high cholesterol and triglyceride levels. This breakthrough could pave the way for new treatments for...
Ex Vivo Gene Correction of Stem Leydig Cells as a Therapeutic Strategy for Hereditary Primary Hypogonadism
Leydig cells (LCs), located in the interstitial compartment of the testes among the seminiferous tubules, are primarily responsible for testosterone production, which is crucial for maintaining the masculine phenotype, endocrine homeostasis, and reproductive function....
Enhancing Diabetic Wound Healing Through Local Silencing of PHD2 and Activation of the AMPK Pathway
Recent data from the International Diabetes Federation (IDF) indicates that in 2017, approximately 425 million people globally were diagnosed with diabetes, with China accounting for 114 million of these cases, the highest number worldwide. One of the main...
Nanodecoys for Antiviral Applications Against SARS-CoV-2 and Other Viruses
The COVID-19 pandemic caused by SARS-CoV-2 has driven the exploration of various mitigation strategies, including vaccines, antiviral drugs, and virus-neutralizing antibodies. However, the emergence of numerous viral variants has limited the effectiveness of these...
AAV CRISPR/Cas9 Gene Editing: A New Horizon in Huntington’s Disease Treatment
Recent advancements in gene therapy have unveiled new possibilities for treating Huntington’s Disease (HD), a neurodegenerative disorder characterized by the progressive decline of motor and cognitive functions. This condition is caused by the expansion of CAG repeats...
Investigating Opioid Peptides: AAV Vector-Delivered Fluorescence Sensors for Studying Opioid Dynamics
Neuropeptides (NPs) play a crucial role in the nervous system, influencing neural activity, brain states, and blood flow. However, the complexity of these molecules, particularly opioid peptides, has presented significant challenges in research due to the lack of...
Exploring Liver Regeneration: A Spatiotemporal Atlas of Mouse Liver
Understanding liver regeneration is crucial for advancements in regenerative medicine and disease research. In a groundbreaking study, researchers combined Stereo-seq (spatiotemporal enhanced resolution omics-sequencing) with single-cell RNA sequencing to create a...
Overview of Key AAV Analytical Testing for Gene Therapy Development
In the development of AAV-based gene therapies, ensuring the quality and efficacy of the viral vectors is paramount. Several critical tests are employed to assess various aspects of AAV vectors, each addressing specific factors that impact the safety and effectiveness...
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