Pigs have substantial potential as biomedical models for studying human developmental
processes, congenital diseases, and pathogen response mechanisms in addition to utility as …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

Objective Infantile‐onset spinal muscular atrophy (SMA) is the most common genetic cause
of infant mortality, typically resulting in death preceding age 2. Clinical trials in this …

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 type 1 (SMA1) is a debilitating neurodegenerative disease resulting
from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec …

Multiple neurodegenerative diseases are characterized by single-protein dysfunction and
aggregation. Treatment strategies for these diseases have often targeted downstream …

Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes | Pediatrics | American
Academy of Pediatrics Skip to Main Content Disclaimer » Advertising AAP logo Search Close …

Spinal muscular atrophy (SMA) is a hereditary neurodegenerative disease with severity
ranging from progressive infantile paralysis and premature death (type I) to limited motor …

The natural history of SMA is complex and variable. For this reason, clinical subgroups have
been defined based upon best motor function attainment during development. Type 1 SMA …

Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …