Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice - PubMed
4 hours ago
- #SOD1-ASOs
- #Intrathecal Injection
- #ALS
- Lumbar intrathecal (IT) injection of SOD1-ASOs provides precise CNS targeting in SOD1-G93A ALS mice.
- IT administration offers minimally invasive, direct drug delivery to cerebrospinal fluid (CSF).
- Acute needle puncture technique in mice is reproducible and less invasive than catheterization.
- Effective downregulation of mutant SOD1 expression and disease phenotype improvement observed.
- Comparable efficacy to intracerebroventricular (ICV) administration with strong translational relevance.
- Technique mirrors human clinical procedures, aiding preclinical CNS therapeutic evaluation.