275 related articles for article (PubMed ID: 24400925)
1. Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA).
Zanetta C; Riboldi G; Nizzardo M; Simone C; Faravelli I; Bresolin N; Comi GP; Corti S
J Cell Mol Med; 2014 Feb; 18(2):187-96. PubMed ID: 24400925
[TBL] [Abstract][Full Text] [Related]
2. Antisense oligonucleotides for the treatment of spinal muscular atrophy.
Porensky PN; Burghes AH
Hum Gene Ther; 2013 May; 24(5):489-98. PubMed ID: 23544870
[TBL] [Abstract][Full Text] [Related]
3. Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.
Sintusek P; Catapano F; Angkathunkayul N; Marrosu E; Parson SH; Morgan JE; Muntoni F; Zhou H
PLoS One; 2016; 11(5):e0155032. PubMed ID: 27163330
[TBL] [Abstract][Full Text] [Related]
4. Established Stem Cell Model of Spinal Muscular Atrophy Is Applicable in the Evaluation of the Efficacy of Thyrotropin-Releasing Hormone Analog.
Ohuchi K; Funato M; Kato Z; Seki J; Kawase C; Tamai Y; Ono Y; Nagahara Y; Noda Y; Kameyama T; Ando S; Tsuruma K; Shimazawa M; Hara H; Kaneko H
Stem Cells Transl Med; 2016 Feb; 5(2):152-63. PubMed ID: 26683872
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. Normalization of Patient-Identified Plasma Biomarkers in SMNĪ7 Mice following Postnatal SMN Restoration.
Arnold WD; Duque S; Iyer CC; Zaworski P; McGovern VL; Taylor SJ; von Herrmann KM; Kobayashi DT; Chen KS; Kolb SJ; Paushkin SV; Burghes AH
PLoS One; 2016; 11(12):e0167077. PubMed ID: 27907033
[TBL] [Abstract][Full Text] [Related]
7. Therapy development for spinal muscular atrophy in SMN independent targets.
Tsai LK
Neural Plast; 2012; 2012():456478. PubMed ID: 22701806
[TBL] [Abstract][Full Text] [Related]
8. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Meyer K; Ferraiuolo L; Schmelzer L; Braun L; McGovern V; Likhite S; Michels O; Govoni A; Fitzgerald J; Morales P; Foust KD; Mendell JR; Burghes AH; Kaspar BK
Mol Ther; 2015 Mar; 23(3):477-87. PubMed ID: 25358252
[TBL] [Abstract][Full Text] [Related]
9. Modeling the differential phenotypes of spinal muscular atrophy with high-yield generation of motor neurons from human induced pluripotent stem cells.
Lin X; Li JJ; Qian WJ; Zhang QJ; Wang ZF; Lu YQ; Dong EL; He J; Wang N; Ma LX; Chen WJ
Oncotarget; 2017 Jun; 8(26):42030-42042. PubMed ID: 28159932
[TBL] [Abstract][Full Text] [Related]
10. Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.
Hua Y; Sahashi K; Rigo F; Hung G; Horev G; Bennett CF; Krainer AR
Nature; 2011 Oct; 478(7367):123-6. PubMed ID: 21979052
[TBL] [Abstract][Full Text] [Related]
11. SMN deficiency in severe models of spinal muscular atrophy causes widespread intron retention and DNA damage.
Jangi M; Fleet C; Cullen P; Gupta SV; Mekhoubad S; Chiao E; Allaire N; Bennett CF; Rigo F; Krainer AR; Hurt JA; Carulli JP; Staropoli JF
Proc Natl Acad Sci U S A; 2017 Mar; 114(12):E2347-E2356. PubMed ID: 28270613
[TBL] [Abstract][Full Text] [Related]
12. Molecular mechanisms and animal models of spinal muscular atrophy.
Edens BM; Ajroud-Driss S; Ma L; Ma YC
Biochim Biophys Acta; 2015 Apr; 1852(4):685-92. PubMed ID: 25088406
[TBL] [Abstract][Full Text] [Related]
13. Spinal Muscular Atrophy and Common Therapeutic Advances.
Bozorg Qomi S; Asghari A; Salmaninejad A; Mojarrad M
Fetal Pediatr Pathol; 2019 Jun; 38(3):226-238. PubMed ID: 31060440
[TBL] [Abstract][Full Text] [Related]
14. Investigation of New Morpholino Oligomers to Increase Survival Motor Neuron Protein Levels in Spinal Muscular Atrophy.
Ramirez A; Crisafulli SG; Rizzuti M; Bresolin N; Comi GP; Corti S; Nizzardo M
Int J Mol Sci; 2018 Jan; 19(1):. PubMed ID: 29316633
[TBL] [Abstract][Full Text] [Related]
15. Metabolic Dysfunction in Spinal Muscular Atrophy.
Deguise MO; Chehade L; Kothary R
Int J Mol Sci; 2021 May; 22(11):. PubMed ID: 34072857
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Disease mechanisms and therapeutic approaches in spinal muscular atrophy.
Tisdale S; Pellizzoni L
J Neurosci; 2015 Jun; 35(23):8691-700. PubMed ID: 26063904
[TBL] [Abstract][Full Text] [Related]
18. Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.
Nizzardo M; Simone C; Dametti S; Salani S; Ulzi G; Pagliarani S; Rizzo F; Frattini E; Pagani F; Bresolin N; Comi G; Corti S
Sci Rep; 2015 Jun; 5():11746. PubMed ID: 26123042
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. Astrocytes influence the severity of spinal muscular atrophy.
Rindt H; Feng Z; Mazzasette C; Glascock JJ; Valdivia D; Pyles N; Crawford TO; Swoboda KJ; Patitucci TN; Ebert AD; Sumner CJ; Ko CP; Lorson CL
Hum Mol Genet; 2015 Jul; 24(14):4094-102. PubMed ID: 25911676
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
