Phosphodiesterases (PDEs), enzymes that degrade 3′, 5′-cyclic nucleotides, are being
pursued as therapeutic targets for several diseases, including those affecting the nervous …

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

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 …

In vivo therapeutic gene transfer has emerged as a novel class of medicines. Its feasibility
relies on the safe and efficacious delivery of genetic cargo to the appropriate targets. The …

Inherited retinal dystrophies and optic neuropathies cause chronic disabling loss of visual
function. The development of recombinant adeno-associated viral vectors (rAAV) gene …

Inner ear gene therapy using adeno-associated viral vectors (AAV) promises to alleviate
hearing and balance disorders. We previously established the benefits of Anc80L65 in …

Owing to their small size and safety profiles, adeno-associated viruses (AAVs) have become
the vector of choice for gene therapy applications in the retina. In addition to the naturally …

An estimated 466 million people suffer from hearing loss worldwide. Sensorineural hearing
loss is characterized by degeneration of key structures of the sensory pathway in the …

Adeno-associated viral vectors (AAVs) have demonstrated potential in applications for
neurologic disorders, and the discovery that some AAVs can cross the blood-brain barrier …

Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus
(AAV)‐based vectors the leading gene delivery vehicles in gene therapy. Pharmaceutical …