Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …

Mucopolysaccharidosis type IVA (MPS IVA, or Morquio syndrome type A) is an inherited
metabolic lysosomal disease caused by the deficiency of the N-acetylglucosamine-6-sulfate …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …

Post‐translational modifications in viral capsids are known to fine‐tune and regulate several
aspects of the infective life cycle of several viruses in the host. Recombinant viruses that are …

Highlights•Innate immune responses to AAV can occur on both short and long timescales.•
Conserved epitopes on different AAV serotypes cloak them from NABs when mutated.•A …

The past several years have witnessed significant advances in the development of
therapeutic gene delivery for neurological disorders of the central nervous system (CNS). In …

There is a strong and growing interest in the development and production of gene therapy
products, including those utilizing adeno‐associated virus (AAV) particles. This is evident …

Since the revolutionary discovery of the CRISPR-Cas technology for programmable genome
editing, its range of applications has been extended by multiple biotechnological tools that …

The Central Nervous System (CNS) is vulnerable to a range of diseases, including
neurodegenerative and oncological conditions, which present significant treatment …