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Human Gene Therapy|April 26, 2019
An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIAAnna L Gray, Claire O'Leary, Aiyin Liao, et al.
Molecular Genetics and Metabolism|March 8, 2024
Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatmentTeresa H Y Wu, Heather A Brown, Heather J Church, et al.
Toxicological Sciences : an Official Journal of the Society of Toxicology|April 2, 2009
Current and future application of genetic toxicity assays: the role and value of in vitro mammalian assaysRosalie K Elespuru, Rajiv Agarwal, Aisar H Atrakchi, et al.
Molecular Vision|June 30, 2015
Characterization of cells from patient-derived fibrovascular membranes in proliferative diabetic retinopathyLeo A Kim, Lindsay L Wong, Dhanesh S Amarnani, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 11, 2013
Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal diseaseAna Sergijenko, Alexander Langford-Smith, Ai Y Liao, et al.
The New England Journal of Medicine|June 10, 2008
Effects of intensive glucose lowering in type 2 diabetes, Hertzel C Gerstein, Michael E Miller, et al.
The Journal of Steroid Biochemistry and Molecular Biology|March 16, 2016
Sterols and oxysterols in plasma from Smith-Lemli-Opitz syndrome patientsWilliam J Griffiths, Jonas Abdel-Khalik, Peter J Crick, et al.
American Heart Journal|June 25, 2005
Implantable cardioverter defibrillators and cardiac resynchronization therapy in patients with left ventricular dysfunction: randomized trial evidence through 2004Sana M Al-Khatib, Gillian D Sanders, Daniel B Mark, et al.
Molecular Genetics and Metabolism|December 4, 2014
Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapyEveline J Langereis, Naomi van Vlies, Heather J Church, et al.
Gene Therapy|May 14, 2004
Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophyL G Gregory, S N Waddington, M V Holder, et al.
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