Publikationen

AIR-001: A highly potent and precise GalNAc-conjugated RNA base editing treatment for alpha-1 antitrypsin deficiency 

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RNA Editing: A Modality for Precise Introduction of Gain-of-Function Variants Identified by Human Genetics

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Building a Leading RNA Editing Platform for the Treatment of Genetic Diseases

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Optimized RESTORE+ oligonucleotides for an efficacious and safe RNA base editing treatment for Alpha-1 Antitrypsin Deficiency

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Precision RNA base editing with engineered and endogenous effectors

Laura S Pfeiffer, Thorsten Stafforst. doi: 10.1038/s41587-023-01927-0.

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Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides

Merkle T, Merz S, Reautschnig P, Blaha A, Li Q, Vogel P, Wettengel J, Li JB, Stafforst T. Nat Biotechnol 2019, 37:133-138, doi: 10.1038/s41587-019-0013-6, PMID: 30692694.

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Harnessing human ADAR2 for RNA repair – Recoding a PINK1 mutation rescues mitophagy

Wettengel J, Reautschnig P, Geisler S, Kahle PJ, Stafforst T. Nucleic Acids Res 2017, 45:2797-2808, doi: 10.1093/nar/gkw911. PMID: 27907896.

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An RNA-deaminase conjugate selectively repairs point mutations

Stafforst T, Schneider MF. Angew Chem Int Ed Engl 2012, 51:11166-11169, doi: 10.1002/anie.201206489, PMID: 23038402.

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