Article
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Molecular therapy : the journal of the American Society of Gene Therapy - 6 Sept 2023
Powers Samantha, Likhite Shibi, Gadalla Kamal K, Miranda Carlos J, Huffenberger Amy J, Dennys Cassandra, Foust Kevin D, Morales Pablo, Pierson Christopher R, Rinaldi Federica, Perry Stephanie, Bolon Brad, Wein Nicolas, Cobb Stuart, Kaspar Brian K, Meyer Kathrin C
Abstract excerpt
The AAV9 gene therapy vector presented in this study is safe in mice and non-human primates and highly efficacious without causing overexpression toxicity, a major challenge for clinical translation of Rett syndrome gene therapy vectors to date. Our team designed a new truncated methyl-CpG-binding protein 2 (MECP2) promoter allowing widespread expression of MECP2 in mice and non-human primates after a single...
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