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4. The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype. Hosseinibarkooie S; Peters M; Torres-Benito L; Rastetter RH; Hupperich K; Hoffmann A; Mendoza-Ferreira N; Kaczmarek A; Janzen E; Milbradt J; Lamkemeyer T; Rigo F; Bennett CF; Guschlbauer C; Büschges A; Hammerschmidt M; Riessland M; Kye MJ; Clemen CS; Wirth B Am J Hum Genet; 2016 Sep; 99(3):647-665. PubMed ID: 27499521 [TBL] [Abstract][Full Text] [Related]
5. Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice. Strathmann EA; Peters M; Hosseinibarkooie S; Rigo FW; Bennett CF; Zaworski PG; Chen KS; Nothnagel M; Wirth B PLoS One; 2018; 13(9):e0203398. PubMed ID: 30188931 [TBL] [Abstract][Full Text] [Related]
6. Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks. Muinos-Bühl A; Rombo R; Janzen E; Ling KK; Hupperich K; Rigo F; Bennett CF; Wirth B Neurobiol Dis; 2022 Sep; 171():105795. PubMed ID: 35724821 [TBL] [Abstract][Full Text] [Related]
7. AAV9-Stathmin1 gene delivery improves disease phenotype in an intermediate mouse model of spinal muscular atrophy. Villalón E; Kline RA; Smith CE; Lorson ZC; Osman EY; O'Day S; Murray LM; Lorson CL Hum Mol Genet; 2019 Nov; 28(22):3742-3754. PubMed ID: 31363739 [TBL] [Abstract][Full Text] [Related]
8. Improved therapeutic approach for spinal muscular atrophy via ubiquitination-resistant survival motor neuron variant. Rhee J; Kang JS; Jo YW; Yoo K; Kim YL; Hann SH; Kim YE; Kim H; Kim JH; Kong YY J Cachexia Sarcopenia Muscle; 2024 Aug; 15(4):1404-1417. PubMed ID: 38650097 [TBL] [Abstract][Full Text] [Related]
9. Plastin 3 ameliorates spinal muscular atrophy via delayed axon pruning and improves neuromuscular junction functionality. Ackermann B; Kröber S; Torres-Benito L; Borgmann A; Peters M; Hosseini Barkooie SM; Tejero R; Jakubik M; Schreml J; Milbradt J; Wunderlich TF; Riessland M; Tabares L; Wirth B Hum Mol Genet; 2013 Apr; 22(7):1328-47. PubMed ID: 23263861 [TBL] [Abstract][Full Text] [Related]
10. 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]
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14. 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]
15. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Dominguez E; Marais T; Chatauret N; Benkhelifa-Ziyyat S; Duque S; Ravassard P; Carcenac R; Astord S; Pereira de Moura A; Voit T; Barkats M Hum Mol Genet; 2011 Feb; 20(4):681-93. PubMed ID: 21118896 [TBL] [Abstract][Full Text] [Related]
16. Neurocalcin Delta Suppression Protects against Spinal Muscular Atrophy in Humans and across Species by Restoring Impaired Endocytosis. Riessland M; Kaczmarek A; Schneider S; Swoboda KJ; Löhr H; Bradler C; Grysko V; Dimitriadi M; Hosseinibarkooie S; Torres-Benito L; Peters M; Upadhyay A; Biglari N; Kröber S; Hölker I; Garbes L; Gilissen C; Hoischen A; Nürnberg G; Nürnberg P; Walter M; Rigo F; Bennett CF; Kye MJ; Hart AC; Hammerschmidt M; Kloppenburg P; Wirth B Am J Hum Genet; 2017 Feb; 100(2):297-315. PubMed ID: 28132687 [TBL] [Abstract][Full Text] [Related]
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19. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Torres-Benito L; Schneider S; Rombo R; Ling KK; Grysko V; Upadhyay A; Kononenko NL; Rigo F; Bennett CF; Wirth B Am J Hum Genet; 2019 Jul; 105(1):221-230. PubMed ID: 31230718 [TBL] [Abstract][Full Text] [Related]
20. 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] [Next] [New Search]