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Human Gene Therapy|September 23, 2014
Biodistribution studies of polymeric nanoparticles for drug delivery in miceMaria Sofia Falzarano, Elena Bassi, Chiara Passarelli, et al.Human Gene Therapy|September 4, 2014
Lentiviral hematopoietic stem cell gene therapy in inherited metabolic disordersGerard WagemakerHuman Gene Therapy|September 4, 2014
Translational and regulatory challenges for exon skipping therapiesAnnemieke Aartsma-Rus, Alessandra Ferlini, Nathalie Goemans, et al.Human Gene Therapy|November 10, 2020
The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous SystemFrançoise Piguet, Timothée de Saint Denis, Emilie Audouard, et al.Human Gene Therapy|September 19, 2020
Retinal Tropism and Transduction of Adeno-Associated Virus Varies by Serotype and Route of Delivery (Intravitreal, Subretinal, or Suprachoroidal) in RatsIan C Han, Justine L Cheng, Erin R Burnight, et al.Human Gene Therapy|July 15, 2022
Adeno-Associated Virus-Mediated Gene Therapy for Patients' Fibroblasts, Induced Pluripotent Stem Cells, and a Mouse Model of Congenital Adrenal HyperplasiaYasuhiro Naiki, Mami Miyado, Miyuki Shindo, et al.Human Gene Therapy|March 16, 2022
Endobronchial Aerosolized AAV1.SERCA2a Gene Therapy in a Pulmonary Hypertension Pig Model: Addressing the Lung Delivery BottleneckOlympia Bikou, Serena Tharakan, Kelly P Yamada, et al.Human Gene Therapy|February 4, 2016
Gene Therapy Strategies for Alzheimer's Disease: An OverviewSandro Alves, Romain Fol, Nathalie CartierHuman Gene Therapy|January 24, 2016
Conditional Cytotoxic Anti-HIV Gene Therapy for Selectable Cell ModificationHimanshu Garg, Anjali JoshiHuman Gene Therapy|January 22, 2016
Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic AcidemiaElizabeth A Harrington, Jennifer L Sloan, Irini Manoli, et al.Pageof 414