在 Cell Reports 期刊发表了题为Engineered IscB-ωRNA system with improved base editing efficiency for disease correction via single AAV delivery in mice 的研究论文。 该研究成功 ...

Next-generation sequencing allows for critical insights into gene therapy products, which can help streamline and accelerate ...

The IVB-2 subtype demonstrated elevated infectiousness and transgene delivery towards cerebral organoids. Figure 3. Cerebral organoids as models for (A) Parkinson’s disease and (B) AAV capsid ...

Safe and effective gene therapy strategies depend on robust and reliable adeno-associated virus (AAV) mediated therapeutic gene delivery. However, scaling up AAV manufacturing to obtain sufficient ...

Gene therapy has experienced an increasing number of successful human clinical trials, leading to 6 FDA approved products using delivery vectors based on adeno-associated viruses (AAV). These ...

Analogous to drug repurposing, AAV capsids can be used for gene delivery applications other than originally intended. With that in mind, we tested two of the capsids, AAV-F and AAV-S, from our ...

Existing gene therapies have primarily used a small number of naturally occurring AAV vectors limited by low delivery efficiency as well as problems with pre-existing immunity and manufacturability.

Currently, there are six US Food and Drug Administration-approved CAR T products, all of which use viral vectors as a ...

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery in the treatment of various human diseases. During development, AAV in-process samples often yield mixed populations of ...