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Boonying Wassamon

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Articles

Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice

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By Xiupeng Chen, Qing Xie, Sarah Nath, Hong Ma, Yasemin Gunes, Tapan Sharma, Hao Liu, Mengtian Cui, Ailing Du, Mengjia Lu, Sophia Liu, Mengyao Xu, Qin Su, Timothy P. Fitzgibbons, Jinghua Liu, Fang Wan, Ran He, Yijie Ma, Jun Yang, Heather L. Gray-Edwards, Thomas Gallagher, Guangping Gao, Jun Xie, Mojiao Tang, Boonying Wassamon, Joseph Yunxi Wu, Veena Kumanan, Phillip W. L. Tai
| Nature Verified
Abstract Spinal muscular atrophy (SMA) results from a deficiency of the survival motor neuron (SMN) protein. Zolgensma, an adeno-associated virus (AAV)-based SMN1 gene-replacement therapy, is approved for SMA, though its long-term efficacy and safety remain uncertain. This study compares a Zolgensma-like benchmark vector with a 2nd-generation vector featuring a codon-optimized SMN1 transgene under the control of an endogenous SMN1 promoter.
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