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 …

Objective To explore whether the utility of neurofilament light chain (NfL), as a biomarker to
aid amyotrophic lateral sclerosis (ALS) therapy development, would be enhanced by …

Spinal motor neuron (MN) dysfunction is the cause of a number of clinically significant
movement disorders. Despite the recent approval of gene therapeutics targeting these MN …

A number of adeno-associated virus (AAV)-based gene therapy products have entered
clinical development, with a few also reaching marketing approval. However, as our …

Over 15 years after hepatotoxicity was first observed following administration of an adeno-
associated virus (AAV) vector during a hemophilia B clinical trial, recent reports of treatment …

In nonhuman primates (NHPs), adeno-associated virus serotype 9 (AAV9) vectorized gene
therapy can cause asymptomatic microscopic injury to dorsal root ganglia (DRG) and …

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

Dorsal root ganglion (DRG) toxicity has been consistently reported as a potential safety
concern after delivery of adeno-associated viruses (AAVs) containing gene-replacement …

Toxic gain-of-function mutations in superoxide dismutase 1 (SOD1) contribute to
approximately 2%–3% of all amyotrophic lateral sclerosis (ALS) cases. Artificial microRNAs …

Dorsal root ganglia (DRG), trigeminal ganglia (TG), other sensory ganglia, and autonomic
ganglia may be injured by some test article classes, including anti-neoplastic …