Hongene Biotech Corporation’s Post

Researchers at the University of Rochester Medical Center developed a ribozyme-activated mRNA trans-ligation technique that enables the delivery of large genes for treating muscular dystrophies that require the replacement of large proteins. Muscular dystrophies are genetic disorders characterized by muscle weakness and degeneration. Treatment remains a challenge as traditional vectors, such as AAVs, are limited in their ability to carry large genetic payloads. To address this issue, researchers developed StitchR, which utilizes ribozymes to facilitate the trans-ligation of mRNA fragments. The method involves designing two mRNA fragments that can be efficiently delivered into cells using conventional vectors. Each fragment is equipped with a ribozyme that can cleave and ligate the fragments to form a full-length, functional mRNA molecule. Results demonstrated that StitchR could restore the expression of large functional muscle proteins, such as dysferlin and dystrophin, to near endogenous levels. This method opens new avenues for the efficient delivery of large therapeutic genes, potentially leading to more effective and widely applicable treatments. Congratulations to all contributors🎉! Sean Lindley Kadiam C Venkata Subbaiah Douglas M. Anderson Find out more: https://lnkd.in/e9JNkS2S #DMD #musculardystrophy #RNAtherapuetics #genetherapy #drugdelivey #geneticdiseases

Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies

Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies

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