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Human Gene Therapy|November 21, 2013
Treatment of congenital neurotransmitter deficiencies by intracerebral ventricular injection of an adeno-associated virus serotype 9 vectorNi-Chung Lee, Yin-Hsiu Chien, Min-Hsiu Hu, et al.Human Gene Therapy|December 26, 2013
Construction and characterization of E1- and E3-deleted adenovirus vectors expressing two antigens from two separate expression cassettesJuliana C Small, Raj K Kurupati, Xiangyang Zhou, et al.Human Gene Therapy|December 31, 2013
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 geneDolan Sondhi, Emma C Scott, Alvin Chen, et al.Human Gene Therapy|March 23, 2016
Efficient Transduction of Corneal Stroma by Adeno-Associated Viral Serotype Vectors for Implications in Gene Therapy of Corneal DiseasesYi Lu, Jianzhong Ai, Dominic Gessler, et al.Human Gene Therapy|May 21, 2016
Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs DiseaseSubha Karumuthil-Melethil, Sahana Nagabhushan Kalburgi, Patrick Thompson, et al.Human Gene Therapy|April 3, 2016
Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and FunctionRonald A Bush, Yong Zeng, Peter Colosi, et al.Human Gene Therapy|May 15, 2016
Advances in Gene Therapy for Diseases of the EyeLolita Petit, Hemant Khanna, Claudio PunzoHuman Gene Therapy|May 15, 2016
Liver-Specific Allergen Gene Transfer by Adeno-Associated Virus Suppresses Allergic Airway Inflammation in MiceCheng-Chi Chan, Chin-Wen Lai, Chia-Jen Wu, et al.Human Gene Therapy|July 15, 2016
Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced PancreatitisDaniel Gaudet, Erik S Stroes, Julie Méthot, et al.Human Gene Therapy|August 11, 2017
Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine ModelKasun Kodippili, Chady H Hakim, Xiufang Pan, et al.Pageof 414