The F7 p.Val22Ile missense mutation affects splicing and can be counteracted by a compensatory U1snRNA

A factor X variant with engineered activation peptide as innovative by-passing agent for haemophilia

Rescue of a FVIII splicing variant with engineered U1snRNAs

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

Engineered suppressor tRNAs as a novel correction approach for recurrent hemophilia A-causing nonsense mutations

Rescue of a hemophilia A-causing FVIII splicing variant via engineered U1SNRNAS

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

A new type 1 von Willebrand Disease (VWD) characterized by increased clearance of von Willebrand Factor (VWF) due to the heterozygous p.P1127S mutation: clinical phenotype and pathogenic mechanisms

An engineered factor X variant as a novel by-passing agent for hemophilia

Comparative Analysis Of Residual Factor VIII Expression from Recurrent F8 Nonsense Mutations Indicates that Localization in the B- domain Favours Readthrough- mediated Protein Output

Deep molecular mechanisms of F8 exon 19 variants and translational approaches in Hemophilia A

Design of a novel factor IX variant with enhanced procoagulant activity and half-life

Genotype and PK Hemophilia B International Study (GePKHIS) - A progress Report

A next-generation rFVIIa fusion protein with enhanced half-life as a novel by-passing tool in hemophilia

Rational engineering of a novel factor IX albumin fusion protein results in enhanced coagulant activity and pharmacokinetic profile

Spontaneous readthrough over recurrent F8 nonsense mutations is associated with residual factor VIII levels: implications for inhibitor risk?

Design of a novel factor IX albumin fusion protein with enhanced coagulant activity and pharmacokinetic profile

Detection of Residual Factor VIII Levels Reveals the Occurrence of Readthrough Over the Majority of F8 Nonsense Mutations

CRISPR activation on coagulation F7 or F8 promoters potentiate trascriptional activity in the normal and mutated gene context

Rescue of multiple Haemophilia A-causing mutations by a single ExSpeU1: the importance of the genomic context