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Wang, Dan
One or more keywords matched the following items that are connected to Wang, Dan
Item TypeName
Concept Disease Models, Animal
Academic Article Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses.
Academic Article In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.
Academic Article Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALS.
Academic Article Attenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppression.
Academic Article The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse.
Academic Article Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation.
Academic Article Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy.
Academic Article Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis.
Academic Article C9orf72 poly(PR) aggregation in nucleus induces ALS/FTD-related neurodegeneration in cynomolgus monkeys.
Academic Article Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency.
Academic Article BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease.
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  • Disease Models Animal