Development of a Novel Strategy to Treat Spinal Muscular Atrophy

Milagros Risco Quiroz
Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by reduced levels of the survival motor neuron (SMN) protein, which results in degeneration of motor neurons, leading to muscle weakness, and, ultimately, death. To provide an effective therapy for SMA, SMN expression must be restored in motor neurons. The goal of our research is to develop protein-based therapeutics for SMA. Protein transduction domain (PTD), from the human immunodeficiency virus-transactivator of transcription (HIV-TAT), mediates the...
This data repository is not currently reporting usage information. For information on how your repository can submit usage information, please see our documentation.