284 related articles for article (PubMed ID: 20225030)
1. Spinal muscular atrophy.
Vitte J; Attali R; Warwar N; Gurt I; Melki J
Adv Exp Med Biol; 2009; 652():237-46. PubMed ID: 20225030
[TBL] [Abstract][Full Text] [Related]
2. Therapeutics development for spinal muscular atrophy.
Sumner CJ
NeuroRx; 2006 Apr; 3(2):235-45. PubMed ID: 16554261
[TBL] [Abstract][Full Text] [Related]
3. Molecular and functional analysis of intragenic SMN1 mutations in patients with spinal muscular atrophy.
Sun Y; Grimmler M; Schwarzer V; Schoenen F; Fischer U; Wirth B
Hum Mutat; 2005 Jan; 25(1):64-71. PubMed ID: 15580564
[TBL] [Abstract][Full Text] [Related]
4. Spinal muscular atrophies.
Viollet L; Melki J
Handb Clin Neurol; 2013; 113():1395-411. PubMed ID: 23622363
[TBL] [Abstract][Full Text] [Related]
5. Splicing changes in SMA mouse motoneurons and SMN-depleted neuroblastoma cells: evidence for involvement of splicing regulatory proteins.
Huo Q; Kayikci M; Odermatt P; Meyer K; Michels O; Saxena S; Ule J; Schümperli D
RNA Biol; 2014; 11(11):1430-46. PubMed ID: 25692239
[TBL] [Abstract][Full Text] [Related]
6. Spinal muscular atrophy: from gene discovery to clinical trials.
Nurputra DK; Lai PS; Harahap NI; Morikawa S; Yamamoto T; Nishimura N; Kubo Y; Takeuchi A; Saito T; Takeshima Y; Tohyama Y; Tay SK; Low PS; Saito K; Nishio H
Ann Hum Genet; 2013 Sep; 77(5):435-63. PubMed ID: 23879295
[TBL] [Abstract][Full Text] [Related]
7. Therapeutic strategies for spinal muscular atrophy: SMN and beyond.
Bowerman M; Becker CG; Yáñez-Muñoz RJ; Ning K; Wood MJA; Gillingwater TH; Talbot K;
Dis Model Mech; 2017 Aug; 10(8):943-954. PubMed ID: 28768735
[TBL] [Abstract][Full Text] [Related]
8. Spinal muscular atrophy: recent advances and future prospects.
Nicole S; Diaz CC; Frugier T; Melki J
Muscle Nerve; 2002 Jul; 26(1):4-13. PubMed ID: 12115944
[TBL] [Abstract][Full Text] [Related]
9. Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery.
Glascock JJ; Osman EY; Wetz MJ; Krogman MM; Shababi M; Lorson CL
Hum Gene Ther; 2012 Mar; 23(3):330-5. PubMed ID: 22029744
[TBL] [Abstract][Full Text] [Related]
10. New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?
Chen TH
Int J Mol Sci; 2020 May; 21(9):. PubMed ID: 32392694
[TBL] [Abstract][Full Text] [Related]
11. Therapeutic strategies for the treatment of spinal muscular atrophy.
Cherry JJ; Androphy EJ
Future Med Chem; 2012 Sep; 4(13):1733-50. PubMed ID: 22924510
[TBL] [Abstract][Full Text] [Related]
12. A large animal model of spinal muscular atrophy and correction of phenotype.
Duque SI; Arnold WD; Odermatt P; Li X; Porensky PN; Schmelzer L; Meyer K; Kolb SJ; Schümperli D; Kaspar BK; Burghes AH
Ann Neurol; 2015 Mar; 77(3):399-414. PubMed ID: 25516063
[TBL] [Abstract][Full Text] [Related]
13. Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.
Mattis VB; Butchbach ME; Lorson CL
J Neurosci Methods; 2008 Oct; 175(1):36-43. PubMed ID: 18771690
[TBL] [Abstract][Full Text] [Related]
14. A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy.
Monani UR; Pastore MT; Gavrilina TO; Jablonka S; Le TT; Andreassi C; DiCocco JM; Lorson C; Androphy EJ; Sendtner M; Podell M; Burghes AH
J Cell Biol; 2003 Jan; 160(1):41-52. PubMed ID: 12515823
[TBL] [Abstract][Full Text] [Related]
15. SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy.
See K; Yadav P; Giegerich M; Cheong PS; Graf M; Vyas H; Lee SG; Mathavan S; Fischer U; Sendtner M; Winkler C
Hum Mol Genet; 2014 Apr; 23(7):1754-70. PubMed ID: 24218366
[TBL] [Abstract][Full Text] [Related]
16. Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.
Cobb MS; Rose FF; Rindt H; Glascock JJ; Shababi M; Miller MR; Osman EY; Yen PF; Garcia ML; Martin BR; Wetz MJ; Mazzasette C; Feng Z; Ko CP; Lorson CL
Hum Mol Genet; 2013 May; 22(9):1843-55. PubMed ID: 23390132
[TBL] [Abstract][Full Text] [Related]
17. Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.
Shababi M; Glascock J; Lorson CL
Hum Gene Ther; 2011 Feb; 22(2):135-44. PubMed ID: 20804424
[TBL] [Abstract][Full Text] [Related]
18. Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy.
Thurmond J; Butchbach ME; Palomo M; Pease B; Rao M; Bedell L; Keyvan M; Pai G; Mishra R; Haraldsson M; Andresson T; Bragason G; Thosteinsdottir M; Bjornsson JM; Coovert DD; Burghes AH; Gurney ME; Singh J
J Med Chem; 2008 Feb; 51(3):449-69. PubMed ID: 18205293
[TBL] [Abstract][Full Text] [Related]
19. Spinal Muscular Atrophy Therapeutics: Where do we Stand?
d'Ydewalle C; Sumner CJ
Neurotherapeutics; 2015 Apr; 12(2):303-16. PubMed ID: 25631888
[TBL] [Abstract][Full Text] [Related]
20. Activation of RNA metabolism-related genes in mouse but not human tissues deficient in SMN.
Olaso R; Joshi V; Fernandez J; Roblot N; Courageot S; Bonnefont JP; Melki J
Physiol Genomics; 2006 Jan; 24(2):97-104. PubMed ID: 16118268
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]