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Gruntman, Alisha
One or more keywords matched the following items that are connected to Gruntman, Alisha
Item TypeName
Academic Article Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles.
Academic Article Gene transfer in the lung using recombinant adeno-associated virus.
Academic Article Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus.
Concept Dependovirus
Academic Article Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.
Academic Article Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency.
Academic Article Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.
Academic Article Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups.
Academic Article 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
Academic Article The rapidly evolving state of gene therapy.
Academic Article Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans.
Academic Article Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy.
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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  • Dependovirus