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AssigneeAscidian Therapeutics, Inc.
InventorsRebekka Krumbach, Scott Dooley, Akiko Doi, Kirk Burkhart, Jesse Gray, Lingtao Peng, Dennis Wu, Akiko Noma, Kirk Gosik, Shimyn Slomovic, Adam Clemens, Robert Bell
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Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.
A nucleic acid trans-splicing molecule comprising:
Dependencies are inferred from the claim text.