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Journal Abstract Search

248 related items for PubMed ID: 24722206

  • 1. Defining the therapeutic window in a severe animal model of spinal muscular atrophy.
    Robbins KL, Glascock JJ, Osman EY, Miller MR, Lorson CL.
    Hum Mol Genet; 2014 Sep 01; 23(17):4559-68. PubMed ID: 24722206
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  • 2. Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy.
    Glascock JJ, Shababi M, Wetz MJ, Krogman MM, Lorson CL.
    Biochem Biophys Res Commun; 2012 Jan 06; 417(1):376-81. PubMed ID: 22172949
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  • 3. 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 15; 20(4):681-93. PubMed ID: 21118896
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  • 4. 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 15; 23(3):330-5. PubMed ID: 22029744
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  • 5. Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy.
    Reilly A, Deguise MO, Beauvais A, Yaworski R, Thebault S, Tessier DR, Tabard-Cossa V, Hensel N, Schneider BL, Kothary R.
    Gene Ther; 2022 Sep 15; 29(9):544-554. PubMed ID: 35462564
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  • 6. PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.
    Little D, Valori CF, Mutsaers CA, Bennett EJ, Wyles M, Sharrack B, Shaw PJ, Gillingwater TH, Azzouz M, Ning K.
    Mol Ther; 2015 Feb 15; 23(2):270-7. PubMed ID: 25369768
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  • 15. 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; 15(4):1404-1417. PubMed ID: 38650097
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  • 16. Long term peripheral AAV9-SMN gene therapy promotes survival in a mouse model of spinal muscular atrophy.
    Reilly A, Yaworski R, Beauvais A, Schneider BL, Kothary R.
    Hum Mol Genet; 2024 Feb 28; 33(6):510-519. PubMed ID: 38073249
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