Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

By Christian J. Hinderer, Nathan Katz, E. Buza, Cecilia Dyer, Tamara Goode, Peter Bell, L. Richman, James M. Wilson. Human Gene Therapy, 2018

701 citations recorded by the source corpus.

Subfields: Genetics, Surgery

View Paper: https://www.semanticscholar.org/paper/377a428d6afe2fc217927caaef723fe65cb092df

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In Genetics

1. Guangping Gao
2. Wei Zhan
3. Thomas L. Gallagher
4. Jiang-Hui Wang
5. Dominic J. Gessler

Citations in the corpus

• Adeno-associated virus as a delivery vector for gene therapy of human diseases (2024, background): “480 Histo-pathological and clinical changes usually occur but can be delayed, suggesting that toxicity is related to the expression of the transgene rather than the capsid.”