Rescue of a panel of Hemophilia A-causing 5’ss splicing mutations by unique Exon-specific U1snRNA variants

Could targeted gene insertion of factor 9 be a potential durable treatment for Hemophilia B?

Reverting highly frequent f8 nonsense mutations: base and prime editing approach for hemophilia A

RESCUE OF A PANEL OF SPLICING MUTATIONS CAUSING HEMOPHILIA A BY ENGINEERED U1-SNRNAS

Prediction of inhibitor risk in haemophilia A using machine learning

Base and Prime Editors as new correction approaches for recurrent Hemophilia A Nonsense Mutations

Tailored collagen binding confers distinct functional properties to engineered factor IX fusion proteins in hemophilia B mouse model

Engineered tRNAs efficiently suppress CDKL5 premature termination codons

A 5’ SPLICE-SITE MUTATION CAUSING MUCOLIPIDOSIS TYPE III CAN BE EFFICIENTLY RESCUED BY U1 SNRNA-BASED THERAPY

Design of an engineered coagulation factor X as a novel by-passing agent for hemophilia

Rescue of a panel of splicing mutations causing hemophilia A by engineered U1snRNAs

1,3,8-Triazaspiro[4.5]decane Derivatives Inhibit Permeability Transition Pores through a FO-ATP Synthase c Subunit Glu119-Independent Mechanism That Prevents Oligomycin A-Related Side Effects

A novel tailored correction approach for recurrent hemophilia-causing nonsense mutations through AntiCodon-Engineered suppressor tRNAs

Engineered tRNAs as a novel tailored correction approach to restore protein biosynthesis and function from hemophilia-causing nonsense mutations

Whole-Exome Sequencing in a Family with an Unexplained Tendency for Venous Thromboembolism: Multicomponent Prediction of Low-Frequency Variant Deleteriousness and of Individual Protein Interaction

Counteracting the Common Shwachman-Diamond Syndrome-Causing SBDS c.258+2T>C Mutation by RNA Therapeutics and Base/Prime Editing

Translational readthrough at F8 nonsense variants in the factor VIII B domain contributes to residual expression and lowers inhibitor association

New Genome Editing Approaches: Base And Prime Editing To Revert Hemophilia A-Causing Point Mutations

Rescue of a FVIII splicing variant with engineered U1snRNAs

Base and Prime editing of DNA as a new therapeutic option for Hemophilia A