Human Gene Therapy

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 Function

Novel Bidirectional Vector Strategy for Amplification of Therapeutic and Reporter Gene Expression

Nonviral Gene Transfer into Liver and Muscle for Treatment of Hyperbilirubinemia in the Gunn Rat

Nonionic Amphiphilic Block Copolymers Promote Gene Transfer to the Lung

Nitroprusside Increases Gene Transfer Associated with Intracoronary Delivery of Adenovirus

Neuroprotective Gene Therapy for Huntington's Disease, Using Polymer-Encapsulated Cells Engineered to Secrete Human Ciliary Neurotrophic Factor: Results of a Phase I Study

Neurodegeneration Augments the Ability of Bone Marrow-Derived Mesenchymal Stem Cells to Fuse with Purkinje Neurons in Niemann-Pick Type C Mice

Monogene and Polygene Therapy for the Treatment of Experimental Prostate Cancers by Use of Apoptotic Genes bax and bad Driven by the Prostate-Specific Promoter ARR2PB

Modulation of Eomes Activity Alters the Size of the Developing Heart: Implications for In Utero Cardiac Gene Therapy

Mitochondrial Gene Therapy Augments Mitochondrial Physiology in a Parkinson's Disease Cell Model

Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients

Low-Level Tyrosine Hydroxylase (TH) Expression Allows for the Generation of Stable TH+ Cell Lines of Human Neural Stem Cells