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3 in total

1. Big win possible for Ionis/Biogen antisense drug in muscular atrophy.

Authors: Ken Garber
Journal: Nat Biotechnol Date: 2016-10-11 Impact factor: 54.908

2. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors: Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal: N Engl J Med Date: 2017-11-02 Impact factor: 91.245

3. Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.

Authors: Christian Hinderer; Nathan Katz; Elizabeth L Buza; Cecilia Dyer; Tamara Goode; Peter Bell; Laura K Richman; James M Wilson
Journal: Hum Gene Ther Date: 2018-02-12 Impact factor: 5.695

3 in total

1 in total

Review 1. Emerging technologies for cystic fibrosis transmembrane conductance regulator restoration in all people with CF.

Authors: Marie E Egan
Journal: Pediatr Pulmonol Date: 2021-02

1 in total