Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
Nicole Armbruster; Annalisa Lattanzi; M. Jeavons; Laetitia van Wittenberghe; Bernard Gjata; Thibaut Marais; Samia Martin; Alban Vignaud · 2016 · Molecular Therapy — Methods & Clinical Development
WASTE classifies this as Failed Experiment Report · AI classification, approximate
An experimental approach did not work as intended — learn what to avoid before investing the same effort.
Abstract (excerpt)
Spinal muscular atrophy (SMA) is an autosomal recessive disease of variable severity caused by mutations in the SMN1 gene. Deficiency of the ubiquitous SMN function results in spinal cord α-motor neuron degeneration and proximal muscle…
Excerpt shown for reference under fair use — read the full paper at the publisher.
About to run something similar?
Run an AI Precheck on your own design to catch failure modes like this one before you spend the time. Your first desk check is free.
Related failures
A Randomized Trial of Intraarterial Treatment for Acute Ischemic Stroke
Negative / Null Result ReportDuodenal Infusion of Donor Feces for Recurrent Clostridium difficile
Negative / Null Result ReportStenting versus Endarterectomy for Treatment of Carotid-Artery Stenosis
Negative / Null Result ReportEffects of Combination Lipid Therapy in Type 2 Diabetes Mellitus
Replication FailureA Randomized Trial of Bevacizumab for Newly Diagnosed Glioblastoma
Negative / Null Result ReportSpironolactone for Heart Failure with Preserved Ejection Fraction
WASTE indexes this work — it does not host or republish it. Failure-type classification is automated and approximate.
Metadata source: OpenAlex · DOI 10.1038/mtm.2016.60