Pages that link to "Q34994553"

The following pages link to Gene Therapeutic Approach Using Mutation-adapted U1 snRNA to Correct a RPGR Splice Defect in Patient-derived Cells (Q34994553):

Displaying 26 items.

• Alternative splicing and retinal degeneration (Q34102442) (← links)
• Identification of Novel and Recurrent Disease-Causing Mutations in Retinal Dystrophies Using Whole Exome Sequencing (WES): Benefits and Limitations (Q36070974) (← links)
• Novel VCAN mutations and evidence for unbalanced alternative splicing in the pathogenesis of Wagner syndrome (Q36611940) (← links)
• Therapeutic activity of modified U1 core spliceosomal particles (Q36768787) (← links)
• Targeting RNA splicing for disease therapy (Q36780738) (← links)
• Ciliary transition zone (TZ) proteins RPGR and CEP290: role in photoreceptor cilia and degenerative diseases (Q37047466) (← links)
• High prevalence of mutations affecting the splicing process in a Spanish cohort with autosomal dominant retinitis pigmentosa (Q37550427) (← links)
• RNA‐based therapeutic approaches for coagulation factor deficiencies (Q37919973) (← links)
• Non-syndromic retinal ciliopathies: translating gene discovery into therapy (Q38030482) (← links)
• Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotides (Q38865127) (← links)
• Localizing the RPGR protein along the cilium: a new method to determine efficacies to treat RPGR mutations (Q38915580) (← links)
• Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations (Q39078944) (← links)
• Mutation-adapted U1 snRNA corrects a splicing error of the dopa decarboxylase gene (Q40569597) (← links)
• An Exon-Specific U1snRNA Induces a Robust Factor IX Activity in Mice Expressing Multiple Human FIX Splicing Mutants. (Q41671484) (← links)
• An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects (Q42156108) (← links)
• An engineered U1 small nuclear RNA rescues splicing defective coagulation F7 gene expression in mice. (Q42224211) (← links)
• Upregulation of functional Kv11.1a isoform expression by modified U1 small nuclear RNA. (Q47608947) (← links)
• Analysis of aberrant pre-messenger RNA splicing resulting from mutations in ATP8B1 and efficient in vitro rescue by adapted U1 small nuclear RNA. (Q51033041) (← links)
• Disease-causing variants of the conserved +2T of 5' splice sites can be rescued by engineered U1snRNAs (Q58580788) (← links)
• Small nuclear RNA-mediated modulation of splicing reveals a therapeutic strategy for a TREM2 mutation and its post-transcriptional regulation (Q88548193) (← links)
• Combining Engineered U1 snRNA and Antisense Oligonucleotides to Improve the Treatment of a BBS1 Splice Site Mutation (Q90200042) (← links)
• Modified U1 snRNA and antisense oligonucleotides rescue splice mutations in SLC26A4 that cause hereditary hearing loss (Q91592890) (← links)
• RNA Therapeutics: How Far Have We Gone? (Q92162442) (← links)
• RNA-Based Therapeutic Strategies for Inherited Retinal Dystrophies (Q92301653) (← links)
• Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges (Q92988365) (← links)
• Molecular Mechanisms and Determinants of Innovative Correction Approaches in Coagulation Factor Deficiencies (Q92989928) (← links)