Breakthroughs and challenges in gene therapy

Gene therapy aims to treat diseases by replacing, inactivating, or introducing genes into patients through a vector — either inside the body (in vivo) or outside of the body (ex vivo). Vectors typically used include nonintegrating viruses such as adeno-associated viruses (AAVs), integrating viruses such as lentiviruses, and non-viral vectors such as lipid nanoparticles (LNPs).

Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body. Therapies are being developed to target areas such as various types of cancer, and autoimmune and rare diseases.

This gene therapy feature shares the latest news and tech developments in cell therapy, RNA therapeutics, and cell line development.

How to characterize partial capsids for a comprehensive assessment of AAV integrity

Accurate quantification of empty and full adeno-associated virus (AAV) capsids is critical for optimizing vectors in gene therapy. In this study, discover the Vericheck ddPCR™ Empty-Full Capsid Kit from Bio-Rad, which is designed to offer a robust and reliable method for assessing capsid integrity, helping to ensure the consistency and effectiveness of AAV-based therapies.

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AAV vectors in gene therapy clinical trials

See how novel assays, like the ELISpot assay from Eurofins Viracor BioPharma Services, are advancing clinical trial screening by providing deeper insights into T cell responses and paving the way for safer and more effective AAV-based gene therapies.

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The future of RNA therapy delivery

Dr. David Bunka shares the benefits of Optimer delivery systems and their potential to overcome targeting issues for improved RNA therapies. Read on to learn their use in a wide variety of applications, including targeted therapies, diagnostics, and research applications.

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Ultrapure water in genetics and genomics research

Explore the vital role of ultrapure water in research, its necessity in ensuring the reliability and reproducibility of experimental results, and how its use can help researchers sidestep potential obstacles.

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Boost lentiviral purity and yield: Comparative insights on M-SAN HQ vs. Benzonase

Discover how M-SAN HQ outperforms Benzonase in lentiviral vector purification. Learn how this advanced nuclease boosts both purity and yield while simplifying the biomanufacturing process.

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