Search Results - "Jalinous, Leila"

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor by Xie, Qing, Chen, Xiupeng, Ma, Hong, Zhu, Yunxiang, Ma, Yijie, Jalinous, Leila, Cox, Gerald F, Weaver, Fiona, Yang, Jun, Kennedy, Zachary, Gruntman, Alisha, Du, Ailing, Su, Qin, He, Ran, Tai, Phillip WL, Gao, Guangping, Xie, Jun

“…Physiological regulation of transgene expression is a major challenge in gene therapy. Onasemnogene abeparvovec ( Zolgensma ® ) is an approved adeno-associated…”
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Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies by Lindley, Sean R., Subbaiah, Kadiam C. Venkata, Priyanka, Fnu, Poosala, Pornthida, Ma, Yijie, Jalinous, Leila, West, Jason A., Richardson, William A., Thomas, Tamlyn N., Anderson, Douglas M.

“…Ribozymes are small catalytic RNA sequences capable of nucleotide-specific self-cleavage found widespread in nature. Ribozyme cleavage generates distinct…”
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