Pages that link to "Q45863479"
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The following pages link to Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing (Q45863479):
Displaying 50 items.
- mRNA trans-splicing in gene therapy for genetic diseases (Q26750650) (← links)
- Gene therapy for hemophilia (Q28081911) (← links)
- Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste (Q28264210) (← links)
- Repair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy (Q28293833) (← links)
- MYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correction (Q28304022) (← links)
- Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice (Q30481282) (← links)
- New high-technology products for the treatment of haemophilia (Q31120359) (← links)
- Exon exchange approach to repair Duchenne dystrophin transcripts (Q33598293) (← links)
- K14 mRNA reprogramming for dominant epidermolysis bullosa simplex. (Q33699579) (← links)
- Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo (Q33713295) (← links)
- Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy (Q33770970) (← links)
- Spliceosome-mediated RNA trans-splicing (Q33990703) (← links)
- Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing (Q34004566) (← links)
- Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies (Q34098340) (← links)
- 5' exon replacement and repair by spliceosome-mediated RNA trans-splicing (Q34365389) (← links)
- Identification of a splicing enhancer in MLH1 using COMPARE, a new assay for determination of relative RNA splicing efficiencies (Q34446453) (← links)
- Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing (Q34514647) (← links)
- Splice-correction strategies for treatment of X-linked agammaglobulinemia. (Q35033817) (← links)
- Trans-splicing repair of mutant p53 suppresses the growth of hepatocellular carcinoma cells in vitro and in vivo (Q35138721) (← links)
- Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing (Q35202462) (← links)
- CD22ΔE12 as a molecular target for corrective repair using RNA trans-splicing: anti-leukemic activity of a rationally designed RNA trans-splicing molecule (Q35216815) (← links)
- Functional repair of p53 mutation in colorectal cancer cells using trans-splicing (Q35273347) (← links)
- Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa (Q35863834) (← links)
- Prospects for gene therapy of haemophilia (Q35824841) (← links)
- Recombinant adeno-associated virus vectors for gene therapy (Q35842668) (← links)
- Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model (Q35847459) (← links)
- Gene therapy of epidermolysis bullosa (Q35873767) (← links)
- Role of bone marrow transplantation for correcting hemophilia A in mice (Q36015228) (← links)
- Gene therapy progress and prospects: reprograming gene expression by trans-splicing (Q36240196) (← links)
- Genetic medicines: treatment strategies for hereditary disorders (Q36423907) (← links)
- Treatment of human disease by adeno-associated viral gene transfer (Q36448393) (← links)
- Cellular and genetic therapies for haemophilia (Q36473038) (← links)
- Genetic engineering for haemophilia A. (Q36600245) (← links)
- Proximity-dependent and proximity-independent trans-splicing in mammalian cells (Q36666307) (← links)
- Modulating the expression of disease genes with RNA-based therapy. (Q36866549) (← links)
- Non-invasive genetic imaging for molecular and cell therapies of cancer (Q37022657) (← links)
- RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice (Q37079038) (← links)
- Tau-based treatment strategies in neurodegenerative diseases (Q37215861) (← links)
- Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing (Q37325074) (← links)
- Replacement of huntingtin exon 1 by trans-splicing (Q37418724) (← links)
- Lessons from non-canonical splicing (Q37499331) (← links)
- Gene therapy for muscular dystrophy: current progress and future prospects. (Q37519650) (← links)
- RNA splicing: disease and therapy (Q37882854) (← links)
- RNA‐based therapeutic approaches for coagulation factor deficiencies (Q37919973) (← links)
- Endothelial progenitor cell-based therapy for hemophilia A (Q37982239) (← links)
- Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy (Q38014583) (← links)
- Hemophilia clinical gene therapy: brief review (Q38076953) (← links)
- Agammaglobulinemia: causative mutations and their implications for novel therapies (Q38161559) (← links)
- New developments in the use of gene therapy to treat Duchenne muscular dystrophy (Q38168863) (← links)
- Trans-splicing improvement by the combined application of antisense strategies. (Q38301824) (← links)