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Base editing rescue of spinal muscular atrophy in cells and in mice.

• Authors : Arbab, Mandana; Matuszek, Zaneta; Kray, Kaitlyn M.

• Source: Science. 4/21/2023, Vol. 380 Issue 6642, p257-257. 1p. 1 Color Photograph.

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SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.

• Authors : Naryshkin, Nikolai A.¹; Weetall, Marla¹; Dakka, Ainal¹

• Source: Science. 8/8/2014, Vol. 345 Issue 6197, p688-693. 6p.

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Plastin 3 Is a Protective Modifier of Autosomal Recessive Spinal Muscular Atrophy.

• Authors : Oprea, Gabriela E.^1,2,3; Kröber, Sandra^1,2; McWhorter, Michelle L.⁴

• Source: Science. 4/25/2008, Vol. 320 Issue 5875, p524-527. 4p. 4 Graphs.

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ERRATA.

• Source: Science. 6/28/2024, Vol. 384 Issue 6703, p1416-1416. 1/9p.

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A SECOND CHANCE: A neuron-targeting gene therapy delivered into the blood saves infants with a deadly spinal disease—and boosts an entire field.

• Authors : Kaiser, Jocelyn

• Source: Science. 11/3/2017, Vol. 358 Issue 6363, p582-585. 4p. 3 Color Photographs.

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Mutations in Dynein Link Motor Neuron Degeneration to Defects in Retrograde Transport.

• Authors : Hafezparast, Majid; Klocke, Rainer; Ruhrberg, Christiana

• Source: Science. 5/2/2003, Vol. 300 Issue 5620, p808-812. 5p. 3 Color Photographs, 1 Graph.

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$2.1 million gene therapy OK’d.

• Source: Science. 5/31/2019, Vol. 364 Issue 6443, p808-809. 2p.

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Identification of Ubiquitin Ligases Required for Skeletal Muscle Atrophy.

• Authors : Bodine, Sue C.; Latres, Esther; Baumhueter, Susanne

• Source: Science. 11/23/2001, Vol. 294 Issue 5547, p1704-1708. 5p. 3 Color Photographs, 9 Black and White Photographs, 2 Graphs.

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De Novo and inherited deletions of the 5q13 region in spinal muscular atrophies.

• Authors : Melki, Judith; Lefebvre, Suzie

• Source: Science. 6/3/1994, Vol. 264 Issue 5164, p1474. 4p. 8 Graphs.

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Antisense rescues babies from killer disease.

• Authors : Wadman, Meredith

• Source: Science. 12/16/2016, Vol. 354 Issue 6318, p1359-1360. 2p. 1 Color Photograph, 2 Diagrams.

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