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AssigneesAscidian Therapeutics, Inc., The Trustees of the University of Pennsylvania
InventorsPhilip R. Johnson, Bruce C. Schnepp, Jean Bennett, Scott J. Dooley, Krishna Jawaharlal Fisher, Junwei Sun
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The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.
A nucleic acid trans-splicing molecule comprising, operatively linked in a 3′-to-5′ direction:
A nucleic acid trans-splicing molecule comprising, operatively linked in a 3′-to-5′ direction:
Dependencies are inferred from the claim text.