Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been
demonstrated to transduce spinal alpha motor neurons when administered intravenously (iv) …

Targeting lower motor neurons (LMNs) for gene delivery could be useful for disorders such
as spinal muscular atrophy and amyotrophic lateral sclerosis. LMNs reside in the ventral …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is
characterized by the deterioration of alpha motor neurons in the brainstem and spinal cord …

Gene therapy for motor neuron diseases requires efficient gene delivery to motor neurons
(MNs) throughout the spinal cord and brainstem. The present study compared adeno …

Spinal muscular atrophy (SMA) is the most frequent lethal genetic neurodegenerative
disorder in infants. The disease is caused by low abundance of the survival of motor neuron …

Adeno-associated virus type 9 (AAV9) is a powerful tool for delivering genes throughout the
central nervous system (CNS) following intravenous injection. Preclinical results in pediatric …

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by mutations in survival
motor neuron 1 (SMN1). Previously, we showed that central nervous system (CNS) delivery …

We have recently demonstrated the remarkable efficiency of self-complementary (sc) AAV9
vectors for central nervous system (CNS) gene transfer following intravenous delivery in …

Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to
administer a therapeutic transgene from the innervated musculature. With the aim of scaling …

Introduction: Adeno-associated viral (AAV) vector-mediated gene delivery to the spinal cord
has finally entered the pathway towards regulatory approval. Phase 1 clinical trials using …