Adeno-associated viruses (AAVs) are the safest and most effective gene delivery vehicles to
drive long-term transgene expression in gene therapy. While animal studies have shown …

Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

Gene therapy using recombinant adeno‐associated virus (rAAV) vectors to treat blinding
retinal dystrophies has become clinical reality. Therapeutically impactful targeting of …

Adeno-associated virus (AAV) has a single-stranded DNA genome encapsidated in a small
icosahedrally symmetric protein shell with 60 subunits. AAV is the leading delivery vector in …

Impairments of vision and hearing are highly prevalent conditions limiting the quality of life
and presenting a major socioeconomic burden. For a long time, retinal and cochlear …

The capsid structures of most Adeno-associated virus (AAV) serotypes, already assigned to
an antigenic clade, have been previously determined. This study reports the remaining …

Inherited retinal degenerations are a leading and untreatbale cause of blindness, and as
such they are targets for gene therapy. Numerous gene therapy treatments have progressed …

All adeno-associated virus (AAV) vectors currently used in clinical trials or approved gene
therapy biologics are based on human or non-human primate AAVs. A major challenge for …

Conclusions: In conclusion, MLIV. K and MLIV. A are AAV peptide display variants selected
in immunocompetent mice with improved hepatocyte tropism and transduction efficiency …

Affinity-based purification of adeno-associated virus (AAV) vectors has replaced density-
based methods for vectors used in clinical settings. This method utilizes camelid single …