Publications by authors named "Stuart G Beattie"

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Long-term effect of gene therapy on Leber's congenital amaurosis.
James W B Bainbridge, Manjit S Mehat, Venki Sundaram, Scott J Robbie, Susie E Barker, Stuart G Beattie

N Engl J Med· May 2015

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Reply to: NGS library preparation may generate artifactual integration sites of AAV vectors.
Christine Kaeppel, Stuart G Beattie, Raffaele Fronza, Richard van Logtenstein, Florence Salmon

Nat Med· June 2014

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Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.
Astrid Pañeda, Esperanza Lopez-Franco, Christine Kaeppel, Carmen Unzu, Ana Gloria Gil-Royo, Stuart G Beattie

Hum Gene Ther· December 2013

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A largely random AAV integration profile after LPLD gene therapy.
Christine Kaeppel, Stuart G Beattie, Raffaele Fronza, Richard van Logtenstein, Florence Salmon

Nat Med· July 2013

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Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates.
Carmen Unzu, Sandra Hervás-Stubbs, Ana Sampedro, Itsaso Mauleón, Uxua Mancheño, Stuart G Beattie

J Transl Med· June 2012

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Adeno-associated virus liver transduction efficiency measured by in vivo [18F]FHBG positron emission tomography imaging in rodents and nonhuman primates.
Astrid Pañeda, Maria Collantes, Stuart G Beattie, Itzia Otano, Jolanda Snapper

Hum Gene Ther· August 2011

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Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancer.
Lucia Vanrell, Marianna Di Scala, Laura Blanco, Itziar Otano, Irene Gil-Farina, Stuart G Beattie

Mol Ther· July 2011

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Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer.
Eduardo Salido, Marisol Rodriguez-Pena, Alfredo Santana, Stuart G Beattie, Harald Petry

Mol Ther· May 2011

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Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.
Carmen Unzu, Ana Sampedro, Itsaso Mauleón, Manuel Alegre, Stuart G Beattie

Mol Ther· February 2011

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Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders.
Astrid Pañeda, Lucia Vanrell, Itsaso Mauleon, Julien S Crettaz, Pedro Berraondo, Stuart G Beattie

Hum Gene Ther· August 2009

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AAV gene therapy as a means to increase apolipoprotein (Apo) A-I and high-density lipoprotein-cholesterol levels: correction of murine ApoA-I deficiency.
Stefan F C Vaessen, Robert Jan Veldman, Elisabeth M Comijn, Jolanda Snapper, Jeroen A Sierts, Stuart G Beattie

J Gene Med· August 2009

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Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice.
Stuart G Beattie, Eric Goetzman, Qiuishi Tang, Thomas Conlon, Martha Campbell-Thompson

J Gene Med· October 2008

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Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD.
Stuart G Beattie, Eric Goetzman, Thomas Conlon, Sean Germain, Glenn Walter

Hum Gene Ther· June 2008

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In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.
Pedro E Cruz, Christian Mueller, Travis L Cossette, Alexandra Golant, Qiushi Tang, Stuart G Beattie

Lab Invest· September 2007

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Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy.
Rahul N Kanadia, Jihae Shin, Yuan Yuan, Stuart G Beattie, Thurman M Wheeler

Proc Natl Acad Sci U S A· August 2006

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Novel tools for production and purification of recombinant adeno-associated viral vectors.
Julian D Harris, Stuart G Beattie, J George Dickson

Methods Mol Med· April 2003

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