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Brian K. Kaspar
Researcher at Nationwide Children's Hospital
Publications - 152
Citations - 12773
Brian K. Kaspar is an academic researcher from Nationwide Children's Hospital. The author has contributed to research in topics: Amyotrophic lateral sclerosis & Spinal muscular atrophy. The author has an hindex of 49, co-authored 148 publications receiving 10898 citations. Previous affiliations of Brian K. Kaspar include Ohio State University & Salk Institute for Biological Studies.
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Journal ArticleDOI
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Jerry R. Mendell,Samiah Al-Zaidy,Richard Shell,W. Dave Arnold,Louise R. Rodino-Klapac,Thomas W. Prior,Linda Lowes,Lindsay N. Alfano,K. Berry,Kathleen Church,John T. Kissel,Sukumar Nagendran,James L’Italien,Douglas M. Sproule,Courtney Wells,Jessica A. Cardenas,Marjet D. Heitzer,Allan Arman Kaspar,Sarah Corcoran,Lyndsey Braun,Shibi Likhite,Carlos Henrique Miranda,Kathrin Meyer,K.D. Foust,Arthur H.M. Burghes,Brian K. Kaspar +25 more
TL;DR: In patients with SMA1, a single intravenous infusion of adenoviral vector containing DNA coding for SMN resulted in longer survival, superior achievement of motor milestones, and better motor function than in historical cohorts.
Journal ArticleDOI
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Kevin D. Foust,Emily Nurre,Emily Nurre,Chrystal L. Montgomery,Anna Hernandez,Curtis M Chan,Brian K. Kaspar,Brian K. Kaspar +7 more
TL;DR: It is shown that adeno-associated virus (AAV) 9 injected intravenously bypasses the BBB and efficiently targets cells of the central nervous system (CNS) and may enable the development of gene therapies for a range of neurodegenerative diseases.
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Astrocytes from familial and sporadic ALS patients are toxic to motor neurons
Amanda M. Haidet-Phillips,Mark E. Hester,Carlos Henrique Miranda,Kathrin Meyer,Lyndsey Braun,Ashley E. Frakes,Ashley E. Frakes,SungWon Song,SungWon Song,Shibi Likhite,Shibi Likhite,Matthew J. Murtha,Matthew J. Murtha,Kevin D. Foust,Meghan Rao,Amy Eagle,Anja Kammesheidt,Ashley Christensen,Jerry R. Mendell,Jerry R. Mendell,Arthur H.M. Burghes,Brian K. Kaspar +21 more
TL;DR: It is demonstrated that astrocytes derived from postmortem tissue from both FALS and SALS patients are similarly toxic to motor neurons and that SOD1 is a viable target for SALS, as its knockdown significantly attenuatesAstrocyte-mediated toxicity toward motor neurons.
Journal ArticleDOI
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Kevin D. Foust,Xueyong Wang,Vicki L. McGovern,Lyndsey Braun,Adam K. Bevan,Adam K. Bevan,Amanda M. Haidet,Amanda M. Haidet,Thanh Le,Pablo R Morales,Mark M. Rich,Arthur H.M. Burghes,Brian K. Kaspar,Brian K. Kaspar +13 more
TL;DR: This work uses scAAV9-mediated postnatal day 1 vascular gene delivery to replace SMN in SMA pups and rescue motor function, neuromuscular physiology and life span and demonstrates the clinical potential of scAAv9 gene therapy for SMA.
Journal ArticleDOI
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.
TL;DR: A directed evolution approach involving the generation of large mutant capsid libraries and selection of adeno-associated virus (AAV) 2 variants with enhanced properties is applied, directing viral evolution to generate 'designer' gene delivery vectors with specified, enhanced properties.