Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …

CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

Abstract SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …

Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …

Importance This ongoing study assesses long-term safety and durability of response in
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …

Background Spinal muscular atrophy is a rare, autosomal recessive, neuromuscular disease
caused by biallelic loss of the survival motor neuron 1 (SMN1) gene, resulting in motor …

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) gene therapies are highly promising, such as the
onasemnogene abeparvovec (Zolgensma) in spinal muscle atrophy (SMA). We report the …

Amyotrophic lateral sclerosis (ALS) is an intractable disease that causes respiratory failure
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …

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