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History (12)
Phase I Study of the Investigational NEDD8-Activating Enzyme Inhibitor Pevonedistat (TAK-924/MLN4924) in Patients with Advanced Solid Tumors.
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway.
A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.
Intrathecal gene therapy for neurologic disease in humans.
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Intrathecal gene therapy for neurologic disease in humans.
Flotte TR. Intrathecal gene therapy for neurologic disease in humans. Mol Ther. 2024 05 01; 32(5):1185-1186.
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PubMed
subject areas
Animals
Genetic Therapy
Genetic Vectors
Humans
Injections, Spinal
Nervous System Diseases
authors with profiles
Terence R Flotte MD