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BEIJING, May 17, 2022 /PRNewswire/ -- Beihai Kangcheng Pharmaceutical .
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In a mouse model of SMA, novel second-generation gene therapy demonstrated efficacy over baseline vehicles on several endpoints, including increased lifespan, restoration of muscle function, and better innervation of the neuromuscular junction, with no acceptance Hepatotoxicity exhibited by baseline vehicle-treated anima.
Finally, SMN1 expression was higher in the central nervous system and lower in peripheral tissues in SMA mice treated with the second-generation gene therapy than in baseline vehicle-treated mice, in a pattern similar to healthy vehicle mi.
This is the first data obtained from Beihai Kangcheng's gene therapy research in cooperation with the Horae Gene Therapy Center Gao L.
"Our novel second-generation gene therapy vector differs from benchmark vectors in the genetic engineering optimization of SMN1 codons under the control of the endogenous SMN1 promot.
Co-director of the Li Weibo Institute for Rare Diseases, director of the Horae Gene Therapy Center and Center for Viral Vectors, Professor of Microbiology and Physiological Systems at the UMass Chan School of Medicine, Penelope Booth Rockwell Research Professor of Biomedical Sciences, and lead author of the study, Guangping Gao, .
forward-looking statements
Forward-looking statements made herein relate only to events or information as of the date on which they are ma.
Source: Beihai Kangcheng