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 viruses (AAVs) are commonly used gene delivery vehicles
for neuroscience research. They have two engineerable features: the capsid (outer protein …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

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 …

Muscle-directed gene therapy with adeno-associated viral (AAV) vectors is undergoing
clinical development for treating neuromuscular disorders and for systemic delivery of …

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

Recombinant adeno-associated virus (rAAV) is the leading platform for delivering genetic
constructs in vivo. To date, three AAV-based gene therapeutic agents have been approved …

In the last four decades, monoclonal antibodies and their derivatives have emerged as a
powerful class of therapeutics, largely due to their exquisite targeting specificity. Several …

We are in an emerging era of gene-based therapeutics with significant promise for rare
genetic disorders. The potential is particularly significant for genetic central nervous system …

GENE THERAPY HAS secured its place in the pharmaceutical industry because it can
address many diseases that have historically been considered untreatable. For the past …