676 results on '"Bennett, C. Frank"'
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2. Opportunities and challenges for innovative and equitable healthcare
3. Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial
4. Author Correction: Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial
5. Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
6. Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
7. Restoration of early postnatal synaptic dysregulation rescues polyglutamine-mediated motor neuron degeneration in mice
8. GOLGA8 increases bulk antisense oligonucleotide uptake and activity in mammalian cells
9. Personalized antisense oligonucleotides ‘for free, for life’ — the n-Lorem Foundation
10. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
11. Antisense Oligonucleotide Therapies for Neurodegenerative Diseases
12. Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration
13. Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS
14. Tool to Resolve Distortions in Elemental and Isotopic Imaging.
15. Safety, tolerability and antiviral activity of the antisense oligonucleotide bepirovirsen in patients with chronic hepatitis B: a phase 2 randomized controlled trial
16. Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells
17. Re-Activation of Neurogenic Niches in Aging Brain
18. Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome
19. Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs
20. Modulation of LMNA splicing as a strategy to treat prelamin A diseases
21. Deletion or Inhibition of the Oxygen Sensor PHD1 Protects against Ischemic Stroke via Reprogramming of Neuronal Metabolism
22. Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation
23. Author Correction: Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer’s disease: a phase 1b, randomized, placebo-controlled trial
24. Synthetic CRISPR RNA-Cas9–guided genome editing in human cells
25. Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
26. Delivery of GalNAc-Conjugated Splice-Switching ASOs to Non-hepatic Cells through Ectopic Expression of Asialoglycoprotein Receptor
27. Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy
28. Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration
29. List of contributors
30. Antisense oligonucleotide drugs for neurological and neuromuscular disease
31. RNA-Targeted Therapeutics
32. MaTAR25 lncRNA regulates the Tensin1 gene to impact breast cancer progression
33. Selective tissue targeting of synthetic nucleic acid drugs
34. SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage
35. Activating the synthesis of progerin, the mutant prelamin A in Hutchinson–Gilford progeria syndrome, with antisense oligonucleotides
36. Supplementary Data from ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth
37. Supplementary Table from ASO-Based PKM Splice-Switching Therapy Inhibits Hepatocellular Carcinoma Growth
38. Data from hsa-miR-191 Is a Candidate Oncogene Target for Hepatocellular Carcinoma Therapy
39. Supplementary Figures 1-9, Table 1 from hsa-miR-191 Is a Candidate Oncogene Target for Hepatocellular Carcinoma Therapy
40. Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies
41. Author Correction: Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome
42. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
43. Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models
44. Long-Term SMN- and Ncald-ASO Combinatorial Therapy in SMA Mice and NCALD-ASO Treatment in hiPSC-Derived Motor Neurons Show Protective Effects
45. Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy
46. Antisense Oligonucleotide-Based Therapies for Diseases Caused by pre-mRNA Processing Defects
47. Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies
48. Translating Antisense Technology into a Treatment for Huntington’s Disease
49. Antisense drugs for rare and ultra-rare genetic neurological diseases.
50. Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
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