Literature DB >> 20212484

Genetic therapy for spinal muscular atrophy.

Alex MacKenzie.   

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Year:  2010        PMID: 20212484     DOI: 10.1038/nbt0310-235

Source DB:  PubMed          Journal:  Nat Biotechnol        ISSN: 1087-0156            Impact factor:   54.908


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  11 in total

1.  Determination of SMN1/SMN2 gene dosage by a quantitative genotyping platform combining capillary electrophoresis and MALDI-TOF mass spectrometry.

Authors:  Hung-Yi Kao; Yi-Ning Su; Hsin-Kai Liao; Ming S Liu; Yu-Ju Chen
Journal:  Clin Chem       Date:  2006-01-26       Impact factor: 8.327

Review 2.  Self-complementary AAV vectors; advances and applications.

Authors:  Douglas M McCarty
Journal:  Mol Ther       Date:  2008-08-05       Impact factor: 11.454

Review 3.  Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies.

Authors:  Nathalie Clément; David R Knop; Barry J Byrne
Journal:  Hum Gene Ther       Date:  2009-08       Impact factor: 5.695

4.  A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells.

Authors:  Richard H Smith; Justin R Levy; Robert M Kotin
Journal:  Mol Ther       Date:  2009-06-16       Impact factor: 11.454

5.  Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction.

Authors:  Jonathan D Finn; Daniel Hui; Harre D Downey; Danielle Dunn; Gary C Pien; Federico Mingozzi; Shangzhen Zhou; Katherine A High
Journal:  Mol Ther       Date:  2009-11-10       Impact factor: 11.454

6.  Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition.

Authors:  Heather L Narver; Lingling Kong; Barrington G Burnett; Dong W Choe; Marta Bosch-Marcé; Addis A Taye; Michael A Eckhaus; Charlotte J Sumner
Journal:  Ann Neurol       Date:  2008-10       Impact factor: 10.422

7.  Assessment of liquid microbead arrays for the screening of newborns for spinal muscular atrophy.

Authors:  Robert E Pyatt; David C Mihal; Thomas W Prior
Journal:  Clin Chem       Date:  2007-09-21       Impact factor: 8.327

8.  Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.

Authors:  Kevin D Foust; Emily Nurre; Chrystal L Montgomery; Anna Hernandez; Curtis M Chan; Brian K Kaspar
Journal:  Nat Biotechnol       Date:  2008-12-21       Impact factor: 54.908

9.  Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect.

Authors:  Tatiana O Gavrilina; Vicki L McGovern; Eileen Workman; Thomas O Crawford; Rocky G Gogliotti; Christine J DiDonato; Umrao R Monani; Glenn E Morris; Arthur H M Burghes
Journal:  Hum Mol Genet       Date:  2008-01-04       Impact factor: 6.150

10.  Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Authors:  Kevin D Foust; Xueyong Wang; Vicki L McGovern; Lyndsey Braun; Adam K Bevan; Amanda M Haidet; Thanh T Le; Pablo R Morales; Mark M Rich; Arthur H M Burghes; Brian K Kaspar
Journal:  Nat Biotechnol       Date:  2010-02-28       Impact factor: 68.164

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  2 in total

1.  Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.

Authors:  Monique A Lorson; Lee D Spate; Melissa S Samuel; Clifton N Murphy; Christian L Lorson; Randall S Prather; Kevin D Wells
Journal:  Transgenic Res       Date:  2011-02-25       Impact factor: 2.788

2.  Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway.

Authors:  Faraz Farooq; Francisco Abadía Molina; Jeremiah Hadwen; Duncan MacKenzie; Luke Witherspoon; Matthew Osmond; Martin Holcik; Alex MacKenzie
Journal:  J Clin Invest       Date:  2011-07-25       Impact factor: 14.808

  2 in total

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