641 results on '"Rigo, Frank"'
Search Results
2. The RNA-binding proteins hnRNP H and F regulate splicing of a MYC-dependent HRAS exon in prostate cancer cells
3. Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation
4. Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies.
5. Early activation of cellular stress and death pathways caused by cytoplasmic TDP-43 in the rNLS8 mouse model of ALS and FTD
6. Targeting alternative splicing of fibronectin in human renal proximal tubule epithelial cells with antisense oligonucleotides to reduce EDA+ fibronectin production and block an autocrine loop that drives renal fibrosis
7. Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study
8. Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome.
9. Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment
10. Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation
11. Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
12. Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
13. Non-invasive Imaging of Antisense Oligonucleotides in the Brain via In Vivo Click Chemistry
14. Differential effects of SARM1 inhibition in traumatic glaucoma and EAE optic neuropathies
15. G4C2 Repeat RNA Initiates a POM121-Mediated Reduction in Specific Nucleoporins in C9orf72 ALS/FTD
16. Acute Trem2 reduction triggers increased microglial phagocytosis, slowing amyloid deposition in mice
17. Splice-switching of the insulin receptor pre-mRNA alleviates tumorigenic hallmarks in rhabdomyosarcoma
18. Corticosteroid sensitization drives opioid addiction
19. Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration
20. A novel ‘social contract’ – An attempt to harmonize a sponsor's exploratory research with a clinical study participant's data rights
21. Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS
22. Murine knockin model for progranulin-deficient frontotemporal dementia with nonsense-mediated mRNA decay
23. Investigating 4R tau isoform imbalance as a mediator of astrocyte dysfunction in tauopathies
24. Splice switching an oncogenic ratio of SmgGDS isoforms as a strategy to diminish malignancy
25. Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome
26. Systemic delivery of a DUX4 targeting antisense oligonucleotide to treat Facioscapulohumeral Muscular Dystrophy
27. Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis
28. RNA-binding protein CPEB1 remodels host and viral RNA landscapes
29. Distinct and shared functions of ALS-associated proteins TDP-43, FUS and TAF15 revealed by multisystem analyses.
30. Robust transcriptome-wide discovery of RNA-binding protein binding sites with enhanced CLIP (eCLIP)
31. Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs
32. Modulation of LMNA splicing as a strategy to treat prelamin A diseases
33. G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons
34. Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation
35. Reduction of Kcnt1 is therapeutic in mouse models of SCN1A and SCN8A epilepsy
36. Suppression of proteolipid protein rescues Pelizaeus–Merzbacher disease
37. Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
38. Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
39. A native function for RAN translation and CGG repeats in regulating fragile X protein synthesis
40. Therapeutic inhibition of mTORC2 rescues the behavioral and neurophysiological abnormalities associated with Pten-deficiency
41. Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor
42. G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons.
43. Reduction of nemo-like kinase increases lysosome biogenesis and ameliorates TDP-43–related neurodegeneration
44. Genetic Downregulation of the Metabotropic Glutamate Receptor Type 5 Dampens the Reactive and Neurotoxic Phenotype of Adult ALS Astrocytes
45. G2C4targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in C9orf72 ALS/FTD human neurons
46. Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration
47. Antisense oligonucleotide therapy reduces seizures and extends life span in an SCN2A gain-of-function epilepsy model
48. MaTAR25 lncRNA regulates the Tensin1 gene to impact breast cancer progression
49. SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage
50. Antisense oligonucleotides targeting microRNA binding sites in the GRN mRNA increase progranulin translation: potential therapeutic strategy for frontotemporal dementia
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