The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

The discovery of naturally occurring adeno-associated virus (AAV) isolates in different
animal species and the generation of engineered AAV strains using molecular genetics …

Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …

Efficient and widespread gene transfer is required for successful treatment of Duchenne
muscular dystrophy (DMD). Here, we performed the first clinical trial using a chimeric adeno …

Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …

Parvoviruses, infecting vertebrates and invertebrates, are a family of single-stranded DNA
viruses with small, non-enveloped capsids with T= 1 icosahedral symmetry. A quarter of a …

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …

Sialylated glycans serve as cell surface attachment factors for a broad range of pathogens.
We report an atypical example, where desialylation increases cell surface binding and …

Over the past 5 years, our laboratory has systematically developed a structure-guided library
approach to evolve new adeno-associated virus (AAV) capsids with altered tissue tropism …