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604. Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

The Other Face of Chimeric Antigen Receptors

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

Reconfigurable Long-Range Phonon Dynamics in Optomechanical Arrays

Physical Review Letters
AstronomyPhysics
2014English

A Conjugated Porous Poly-Benzobisthiadiazole Network for a Visible Light-Driven Photoredox Reaction

Journal of Materials Chemistry A
Materials ScienceChemistrythe EnvironmentSustainabilityRenewable Energy
2014English

Eigenpolarizations for Giant Transverse Optical Beam Shifts

Physical Review Letters
AstronomyPhysics
2014English

Leptin: A Biomarker for Sleep Disorders?

Sleep Medicine Reviews
PulmonaryNeurologyRespiratory MedicinePhysiology
2014English

Theoretical Prediction and Spectroscopic Fingerprints of an Orbital Transition inCeCu2Si2

Physical Review Letters
AstronomyPhysics
2014English

Research Highlights

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

Unstable Avoided Crossing in Coupled Spinor Condensates

Physical Review Letters
AstronomyPhysics
2014English

134. Assessment of the Dystrophin Gene Exon 53 Skipping Using DMD Patient-Derived Fibroblasts for Exploratory Clinical Trial of Antisense Drug NS-065/NCNP-01

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2014English

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