Related Experiment Video
Updated: Dec 20, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
Amy G Feldman1, Julie A Parsons1, Cullen M Dutmer1
1Children's Hospital Colorado, University of Colorado School of Medicine, Denver; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
Abstract:
Spinal muscular atrophy is a neurodegenerative disease resulting from irreversible loss of anterior horn cells owing to biallelic deletions/mutations in the survival motor neuron (SMN) 1 gene. Gene replacement therapy using an adeno-associated virus vector containing the SMN gene was approved by the US Food and Drug Administration in May 2019. We report 2 cases of transient, drug-induced liver failure after this therapy.

