Lo U1 snRNP (small nuclear RiboNuclear Particle) e'una particella fondamentale presente
nello spliceosoma ed è coinvolto in diverse fasi del processamento del precursore dell'RNA …

ABSTRACT Small nuclear U1-RNAs (snRNAs), the spliceosome components selectively
recognizing donor splice sites (5'ss), were engineered to restore correct mRNA processing …

Small nuclear U1-RNAs (snRNAs), the spliceosome components selectively recognizing
donor splice sites (5′ ss), were engineered to restore correct mRNA processing in a …

Changes affecting mRNA processing represent a frequent cause of severe coagulation
factor defects and of all inherited human diseases. We extensively investigated the IVS7+ …

Background The ability of the spliceosomal small nuclear RNA U1 (U1snRNA) to rescue pre‐
mRNA splicing impaired by mutations makes it an attractive therapeutic molecule …

Background: The small nuclear RNA U1 (U1snRNA), the component of the spliceosomal
U1snRNP, is an attractive therapeutic molecule because of its ability to rescue mRNA …

Loss-of-function mutations in the human coagulation factor 9 (F9) gene lead to hemophilia
B. Here, we dissected the consequences and the pathomechanism of a non-coding mutation …

Substitutive therapy has significantly ameliorated the quality of life of patients with
coagulation factor deficiencies. However, there are some limitations that support research …

Abstract Abstract 754 Background: The small nuclear RNA U1 (U1snRNA), the component
of the U1snRNP with a key role in pre-mRNA splicing, is an attractive therapeutic molecule …

Background: Limitations of replacement therapy for coagulation deficiencies encourage
research toward alternative strategies. The spliceosomal U1snRNA, having key role in pre …