AAV Immunogenicity: Challenges, Developments, and Innovations
Adeno-associated viruses (AAVs) have emerged as one of the most promising vectors for gene therapy due to their ability to efficiently deliver therapeutic genes to a wide range of tissues, their relatively low immunogenicity compared to other viral vectors, and their...
Riding Momentum in the DMD Space: A New Wave of Progress
Recently, topline results from Part 2 of Sarepta Therapeutics’ EMBARK trial showed significant improvements in motor function among Duchenne muscular dystrophy (DMD) patients who switched from placebo to Elevidys. These findings reinforce the potential of Elevidys to...
AAV-Cas13d for Multiplexed Immunosuppressive Gene Inhibition in Cancer Therapy
Feifei Zhang et al. recently published a research article in Nature Biotechnology titled "Multiplexed inhibition of immunosuppressive genes with Cas13d for combinatorial cancer immunotherapy." This paper describes a novel multiplexed, universal combinatorial...
The Hidden Threat to AAV Purity: Uncovering Ferritin Contamination
Adeno-associated virus (AAV), valued for its low immunogenicity, broad tropism, and long-lasting in vivo gene expression, has emerged as the leading vector for gene therapy delivery. For AAV vectors intended for clinical use, the FDA requires stringent quality control...
Advancing AAV-Based Gene Therapy for Hearing Loss Using Mini-PCDH15 Variants
Hearing loss affects millions of people worldwide and can be caused by genetic defects in key proteins essential for auditory function. Recent research by Pedro De-la-Torre and colleagues (doi: https://doi.org/10.1101/2024.06.16.599132) has provided significant...
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...
Advances in AAV-SB Transposon Hybrid Systems for Liver-Targeted Gene Therapies
*Nicolás Sandoval-Villegas, Zoltán Ivics, The best of both worlds: AAV-mediated gene transfer empowered by LNP delivery of Sleeping Beauty transposase for durable transgene expression in vivo, Molecular Therapy, Volume 32, Issue 10, 2024, Pages 3211-3214, ISSN...
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...
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