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Proceedings of the National Academy of Sciences of the United States of America|February 20, 2014
IgH class switching exploits a general property of two DNA breaks to be joined in cis over long chromosomal distancesMonica Gostissa, Bjoern Schwer, Amelia Chang, et al.
Molecular Therapy. Methods & Clinical Development|January 24, 2018
Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis PigmentosaTimothy K MacLachlan, Mark N Milton, Oliver Turner, et al.
Investigative Ophthalmology & Visual Science|August 18, 2009
Self-complementary AAV virus (scAAV) safe and long-term gene transfer in the trabecular meshwork of living rats and monkeysLaKisha K Buie, Carol A Rasmussen, Eric C Porterfield, et al.
Molecular Therapy. Methods & Clinical Development|December 3, 2019
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIANicolina Cristina Sorrentino, Vincenzo Cacace, Maria De Risi, et al.
Cell|October 4, 2011
Genome-wide translocation sequencing reveals mechanisms of chromosome breaks and rearrangements in B cellsRoberto Chiarle, Yu Zhang, Richard L Frock, et al.
Molecular Therapy. Methods & Clinical Development|June 26, 2023
Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primatesNancy Chen, David E Ehmann, Robert Crooker, et al.
Molecular Therapy. Methods & Clinical Development|July 23, 2015
AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout miceVivian W Choi, Chad E Bigelow, Terri L McGee, et al.
Human Gene Therapy|December 29, 2025
Intravenous Delivery of a Gene Therapy Vector that Expresses an Antitransferrin Receptor 1 Nanobody-I2S Fusion Protein Rescued Central Nervous System Lysosomal Burden in Mucopolysaccharidosis II MiceWanida Ruangsiriluk, Sarah Melissa P Jacobo, Andrea Iskenderian, et al.
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