Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo
gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer …

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 …

Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101;
ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver …

The neurodegenerative disease spinal muscular atrophy (SMA) is caused by deficiency in
the survival motor neuron (SMN) protein. Currently approved SMA treatments aim to restore …

The field of gene therapy is striving more than ever to define a path to the clinic and the
market. Twenty gene therapy products have already been approved and over two thousand …

Twenty-five years ago, the underlying genetic cause for one of the most common and
devastating inherited diseases in humans, spinal muscular atrophy (SMA), was identified …

Spinal muscular atrophy (SMA) is a heritable neuromuscular disorder that causes
degeneration of the alpha motor neurons from anterior horn cells in the spinal cord, which …

Introduction: Spinal muscular atrophy (SMA) is one of the most common inherited
neuromuscular disorders. It causes progressive muscle weakness and results in significant …