Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Guangping Gao, Mauricio R. Alvira, Lili Wang, +3

Proceedings of the National Academy of Sciences

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

William W. Hauswirth, Tomás S. Alemán, Shalesh Kaushal, +8

Human Gene Therapy

Inhibition of glycogen synthase kinase-3 by lithium correlates with reduced tauopathy and degeneration<i>in vivo</i>

Wendy Noble, Emmanuel Planel, Cindy Zehr, +14

Proceedings of the National Academy of Sciences

A Robust Algorithm for Copy Number Detection Using High-Density Oligonucleotide Single Nucleotide Polymorphism Genotyping Arrays

Yasuhito Nannya, Masashi Sanada, Kumi Nakazaki, +8

Cancer Research

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang Yang, Lili Wang, Peter Bell, +9

Nature Biotechnology

Systems BiologyHepatologyCas9-mediated CorrectionLiver PhysiologyInherited Metabolic Disease +8

Cdk5 Is a Key Factor in Tau Aggregation and Tangle Formation In Vivo

Wendy Noble, Vicki Olm, Kazuyuki Takata, +16

Neuron

Tau AggregationProtein FunctionSignal TransductionSignaling PathwayProtein Folding +12

<i>ACVR1</i> ^<i>R206H</i> receptor mutation causes fibrodysplasia ossificans progressiva by imparting responsiveness to activin A

Sarah Hatsell, Vincent Idone, Dana M. Alessi Wolken, +17

Science Translational Medicine

Activin FamilySclerostinCell DeathOsteogenesisOsteoporosis +17

Anesthesia Leads to Tau Hyperphosphorylation through Inhibition of Phosphatase Activity by Hypothermia

Emmanuel Planel, K. Richter, Charles E. Nolan, +10

Journal of Neuroscience

Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery

Masahiko Hoshijima, Yasuhiro Ikeda, Yoshitaka Iwanaga, +10

Nature Medicine

Cardiac MuscleCardiomyopathyHeart FailureCardiovascular DiseaseMedicine +10

Thyroid and Glucocorticoid Hormones Promote Functional T-Tubule Development in Human-Induced Pluripotent Stem Cell–Derived Cardiomyocytes

Shan Parikh, Daniel J. Blackwell, Nieves Gómez‐Hurtado, +9

Circulation Research