Figure 1: Strategy for packaging a large gene by the split AAV vectors through heterodimerization. Figure 3: Characterization of the split AAV vector heterodimerization by RT–PCR and DNA-PCR.

Many viral vectors have shown promise as gene delivery systems in small-animal studies 1. As these vectors progress to the next level of phase I and phase II clinical studies, it is important to ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

Viral vectors introduce genes and gene editing sequences into cells, and are a popular platform for gene therapy due to their safety and efficacy features. Adeno-associated viral (AAV) vectors in ...

Translational projects in the lab involve the development of novel gene delivery vectors and applications. We use Adeno-Associated Viral (AAV) vectors to manipulate gene expression in animal models, ...

To address these challenges, researchers are developing novel viral vector designs, such as self-inactivating vectors, tissue-specific promoters, and targeted gene editing approaches. Additionally, ...

as well as precisely modified adeno-associated virus vectors for human gene therapy; 3) Elucidating the roles of various post-translational modifications of mammalian proteins; 4) Versatile analysis ...

Genethon & Eukarÿs enter strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies: Evry, France Friday, January 24, 2025, ...

Although adeno-associated virus (AAV) vectors can successfully deliver gene-editing agents in vivo, they are associated with several limitations. Therefore, additional delivery methods are needed ...

has demonstrated the successful use of lentiviral vectors to deliver gene therapy for patients with severe hemophilia A. The study presents a potential alternative to adeno-associated virus (AAV ...

Its product candidates include the NAV Technology Platform, which consists of exclusive rights to novel adeno-associated viral vectors, and therapeutic programs such as RGX-314, RGX-202 ...