368 related articles for article (PubMed ID: 27621445)
1. Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
Hammond SM; Hazell G; Shabanpoor F; Saleh AF; Bowerman M; Sleigh JN; Meijboom KE; Zhou H; Muntoni F; Talbot K; Gait MJ; Wood MJ
Proc Natl Acad Sci U S A; 2016 Sep; 113(39):10962-7. PubMed ID: 27621445
[TBL] [Abstract][Full Text] [Related]
2. Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.
Shabanpoor F; Hammond SM; Abendroth F; Hazell G; Wood MJA; Gait MJ
Nucleic Acid Ther; 2017 Jun; 27(3):130-143. PubMed ID: 28118087
[TBL] [Abstract][Full Text] [Related]
3. Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts.
Farrelly-Rosch A; Lau CL; Patil N; Turner BJ; Shabanpoor F
Neurochem Int; 2017 Sep; 108():213-221. PubMed ID: 28389270
[TBL] [Abstract][Full Text] [Related]
4. Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells.
Pagliarini V; Guerra M; Di Rosa V; Compagnucci C; Sette C
J Neurochem; 2020 Apr; 153(2):264-275. PubMed ID: 31811660
[TBL] [Abstract][Full Text] [Related]
5. Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of
Toosaranont J; Ruschadaariyachat S; Mujchariyakul W; Arora JK; Charoensawan V; Suktitipat B; Palmer TN; Fletcher S; Wilton SD; Mitrpant C
Int J Mol Sci; 2022 Apr; 23(7):. PubMed ID: 35409296
[TBL] [Abstract][Full Text] [Related]
6. Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.
Williams JH; Schray RC; Patterson CA; Ayitey SO; Tallent MK; Lutz GJ
J Neurosci; 2009 Jun; 29(24):7633-8. PubMed ID: 19535574
[TBL] [Abstract][Full Text] [Related]
7. Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape.
Wood MJA; Talbot K; Bowerman M
Hum Mol Genet; 2017 Oct; 26(R2):R151-R159. PubMed ID: 28977438
[TBL] [Abstract][Full Text] [Related]
8. Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model.
Sheng L; Rigo F; Bennett CF; Krainer AR; Hua Y
Nucleic Acids Res; 2020 Apr; 48(6):2853-2865. PubMed ID: 32103257
[TBL] [Abstract][Full Text] [Related]
9. Morpholino-Mediated Exon Inclusion for SMA.
Zhou H; Muntoni F
Methods Mol Biol; 2018; 1828():467-477. PubMed ID: 30171560
[TBL] [Abstract][Full Text] [Related]
10. Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.
Rigo F; Chun SJ; Norris DA; Hung G; Lee S; Matson J; Fey RA; Gaus H; Hua Y; Grundy JS; Krainer AR; Henry SP; Bennett CF
J Pharmacol Exp Ther; 2014 Jul; 350(1):46-55. PubMed ID: 24784568
[TBL] [Abstract][Full Text] [Related]
11. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.
Porensky PN; Mitrpant C; McGovern VL; Bevan AK; Foust KD; Kaspar BK; Wilton SD; Burghes AH
Hum Mol Genet; 2012 Apr; 21(7):1625-38. PubMed ID: 22186025
[TBL] [Abstract][Full Text] [Related]
12. Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.
Mitrpant C; Porensky P; Zhou H; Price L; Muntoni F; Fletcher S; Wilton SD; Burghes AH
PLoS One; 2013; 8(4):e62114. PubMed ID: 23630626
[TBL] [Abstract][Full Text] [Related]
13. Discovery of a CNS penetrant small molecule SMN2 splicing modulator with improved tolerability for spinal muscular atrophy.
Ando S; Suzuki S; Okubo S; Ohuchi K; Takahashi K; Nakamura S; Shimazawa M; Fuji K; Hara H
Sci Rep; 2020 Oct; 10(1):17472. PubMed ID: 33060681
[TBL] [Abstract][Full Text] [Related]
14. Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.
Zhao X; Feng Z; Ling KK; Mollin A; Sheedy J; Yeh S; Petruska J; Narasimhan J; Dakka A; Welch EM; Karp G; Chen KS; Metzger F; Ratni H; Lotti F; Tisdale S; Naryshkin NA; Pellizzoni L; Paushkin S; Ko CP; Weetall M
Hum Mol Genet; 2016 May; 25(10):1885-1899. PubMed ID: 26931466
[TBL] [Abstract][Full Text] [Related]
15. Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review.
Nakevska Z; Yokota T
Eur J Cell Biol; 2023 Jun; 102(2):151326. PubMed ID: 37295266
[TBL] [Abstract][Full Text] [Related]
16. Efficient systemic CNS delivery of a therapeutic antisense oligonucleotide with a blood-brain barrier-penetrating ApoE-derived peptide.
Yeoh YQ; Amin A; Cuic B; Tomas D; Turner BJ; Shabanpoor F
Biomed Pharmacother; 2024 Jun; 175():116737. PubMed ID: 38749176
[TBL] [Abstract][Full Text] [Related]
17. Potential of Cell-Penetrating Peptide-Conjugated Antisense Oligonucleotides for the Treatment of SMA.
Leckie J; Yokota T
Molecules; 2024 Jun; 29(11):. PubMed ID: 38893532
[TBL] [Abstract][Full Text] [Related]
18. Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.
Nizzardo M; Simone C; Salani S; Ruepp MD; Rizzo F; Ruggieri M; Zanetta C; Brajkovic S; Moulton HM; Müehlemann O; Bresolin N; Comi GP; Corti S
Clin Ther; 2014 Mar; 36(3):340-56.e5. PubMed ID: 24636820
[TBL] [Abstract][Full Text] [Related]
19. Recent Advances and Clinical Applications of Exon Inclusion for Spinal Muscular Atrophy.
Son HW; Yokota T
Methods Mol Biol; 2018; 1828():57-68. PubMed ID: 30171534
[TBL] [Abstract][Full Text] [Related]
20. Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.
Passini MA; Bu J; Richards AM; Kinnecom C; Sardi SP; Stanek LM; Hua Y; Rigo F; Matson J; Hung G; Kaye EM; Shihabuddin LS; Krainer AR; Bennett CF; Cheng SH
Sci Transl Med; 2011 Mar; 3(72):72ra18. PubMed ID: 21368223
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
