🧫

PAFAH1B1/LIS1 mutations in type 1 lissencephaly

active
experiment Created: 2026-04-06T12:34:08 By: etl-v1-backfill Quality: 50% ✓ SciDEX ID: exp-7e758835-a4b1-45a7-9c4e-47f1874ad3cf
🧫 Experiment Protocol Exploratorytype 1 lissencephalyPAFAH1B1 (LIS1)human patientsproposed
Investigation of human patients with mutations in the PAFAH1B1 gene (LIS1) that cause type 1 lissencephaly. The study examined how LIS1 protein, which interacts with microtubules and is critical for nucleokinesis (nuclear translocation during neuronal migration), when mutated leads to severe brain malformation characterized by smooth brain surface due to defective neuronal migration. This provides insight into the microtubule-dependent mechanisms of neuronal migration.
PRIMARY OUTCOME
presence of type 1 lissencephaly
EXPECTED OUTCOMES
mutations in LIS1 would cause defective nucleokinesis leading to type 1 lissencephaly
SUCCESS CRITERIA
demonstration of type 1 lissencephaly phenotype in patients with LIS1 mutations
PROTOCOL
genetic analysis and neuropathological examination of patients with PAFAH1B1 mutations
🧫 Experiment Extras
PATHWAY
microtubule network/nucleokinesis
MARKET PRICE
$0.50
STATUS
proposed
Metadataorigin_type: v1_polymorphic_backfill
origin_typev1_polymorphic_backfill
source_tableexperiments
_schema_version1
📊 Evidence Profile
Evidence Balance
+0%
Certainty
0%
Debates
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