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 …

Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

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 …

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 …

The main aim of this review is to summarize the current state-of-art in the field of childhood
Neuronal Ceroid Lipofuscinosis (NCL), a group of rare neurodegenerative disorders. These …

Adeno-associated viruses (AAVs) are being increasingly used as gene therapy vectors in
clinical studies especially targeting central nervous system (CNS) disorders …

The blood‐brain barrier (BBB) poses daunting challenges in treating diseases associated
with the central nervous system (CNS). Recently, the traditional notion of the absence of the …