Stuart G. Beattie

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

James Bainbridge, Manjit Mehat, Venki Sundaram et al. · New England Journal of Medicine · 2015 · 713 citations · Full text

Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy

Rahul Kanadia, Jihae Shin, Yuan Yuan et al. · Proceedings of the National Academy of Sciences · 2006 · 362 citations · Full text

Genetics, Disease Gene Identification, Mbnl1 Activity +17

A largely random AAV integration profile after LPLD gene therapy

Christine Kaeppel, Stuart G. Beattie, Raffaele Fronza et al. · Nature Medicine · 2013 · 191 citations · Full text

Gene Therapies, Lpld Gene Therapy, Cell Therapy +7

Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders

Astrid Pañeda, Lucía Vanrell, Itsaso Mauleón et al. · Human Gene Therapy · 2009 · 107 citations

Genetics, Immunology, Pathology +16

Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates

Carmen Unzu, Sandra Hervás‐Stubbs, Ana Sampedro et al. · Journal of Translational Medicine · 2012 · 68 citations · Full text