| P50 | author | Annemieke Aartsma-Rus | Q42215747 |
| P2093 | author name string | Marcel Veltrop | |
| P2860 | cites work | Spinal muscular atrophy | Q21202863 |
| | Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study | Q24616493 |
| | Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration | Q24617850 |
| | Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study | Q24643018 |
| | In-frame deletion in a novel centrosomal/ciliary protein CEP290/NPHP6 perturbs its interaction with RPGR and results in early-onset retinal degeneration in the rd16 mouse | Q24671808 |
| | Regulation of alternative splicing by local histone modifications: potential roles for RNA-guided mechanisms | Q26999239 |
| | Splicing therapy for neuromuscular disease | Q27009581 |
| | Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping | Q28291830 |
| | Lack of exon 10 in the murine tau gene results in mild sensorimotor defects with aging | Q30597043 |
| | Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy | Q33877924 |
| | Systemic administration of PRO051 in Duchenne's muscular dystrophy | Q34172661 |
| | Antisense oligonucleotides for the treatment of spinal muscular atrophy | Q34336279 |
| | Fibrodysplasia ossificans progressiva: mechanisms and models of skeletal metamorphosis | Q34644842 |
| | Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy | Q34695728 |
| | Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressiva | Q34828364 |
| | The domestic cat as a large animal model for characterization of disease and therapeutic intervention in hereditary retinal blindness | Q34961635 |
| | Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model | Q35327028 |
| | Lack of myostatin results in excessive muscle growth but impaired force generation | Q35629418 |
| | AON-mediated Exon Skipping Restores Ciliation in Fibroblasts Harboring the Common Leber Congenital Amaurosis CEP290 Mutation | Q36077787 |
| | Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy. | Q36482835 |
| | Targeting RNA splicing for disease therapy | Q36780738 |
| | An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study | Q37015657 |
| | Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy. | Q37035750 |
| | Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy | Q37105210 |
| | New insights in gene-derived therapy: the example of Duchenne muscular dystrophy | Q37814437 |
| | Splice modulating therapies for human disease | Q37994280 |
| | Spinal muscular atrophy: manifestations and management | Q38015797 |
| | Tau alternative splicing in familial and sporadic tauopathies. | Q38027938 |
| | Genetic abnormalities in fibrodysplasia ossificans progressiva | Q38066358 |
| | Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy. | Q38092179 |
| | Structural analyses of the pre-mRNA splicing machinery | Q38099614 |
| | Peptide nucleic acid (PNA) cell penetrating peptide (CPP) conjugates as carriers for cellular delivery of antisense oligomers | Q38325571 |
| | Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. | Q38340328 |
| | Targeting a pre-mRNA structure with bipartite antisense molecules modulates tau alternative splicing | Q39306317 |
| | A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice | Q39350652 |
| | Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290. | Q39480239 |
| | Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model | Q39880997 |
| | Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. | Q41890992 |
| | Eteplirsen for the treatment of Duchenne muscular dystrophy | Q45855674 |
| | Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse | Q45863474 |
| | Neurodegenerative disorder FTDP-17-related tau intron 10 +16C → T mutation increases tau exon 10 splicing and causes tauopathy in transgenic mice. | Q50744118 |
| | Lack of myostatin impairs mechanical performance and ATP cost of contraction in exercising mouse gastrocnemius muscle in vivo. | Q51028734 |
| | Local dystrophin restoration with antisense oligonucleotide PRO051 | Q80412961 |
| P433 | issue | 1 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 50-55 | |
| P577 | publication date | 2014-01-31 | |
| P1433 | published in | Experimental Cell Research | Q1524289 |
| P1476 | title | Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases | |
| P478 | volume | 325 | |