Therapeutic potential of CERE-110 (AAV2-NGF): Targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons

• Published in: Experimental neurology Vol. 211; no. 2; pp. 574 - 584
• Main Authors: Bishop, Kathie M., Hofer, Eva K., Mehta, Arpesh, Ramirez, Anthony, Sun, Liangwu, Tuszynski, Mark, Bartus, Raymond T.
• Format: Journal Article
• Language: English
• Published: Amsterdam Elsevier Inc 01.06.2008; Elsevier
• Subjects: Adeno-associated virus; Adenoviridae - genetics; Adenoviridae - metabolism; Adult and adolescent clinical studies; Alzheimer's disease; Animals; Basal forebrain cholinergic neurons; Biological and medical sciences; CERE-110; Cholinergic Fibers - metabolism; Cholinergic Fibers - virology; Degenerative and inherited degenerative diseases of the nervous system. Leukodystrophies. Prion diseases; Development. Senescence. Regeneration. Transplantation; Dose–response; Drug Delivery Systems - methods; Fundamental and applied biological sciences. Psychology; Gene delivery; Genetic Vectors - administration & dosage; Genetic Vectors - metabolism; Humans; Male; Marmota; Medical sciences; Nerve Degeneration - drug therapy; Nerve Degeneration - genetics; Nerve Degeneration - prevention & control; Nerve growth factor; Nerve Growth Factor - administration & dosage; Nerve Growth Factor - metabolism; Nerve Growth Factor - therapeutic use; Neurology; Neurotrophin; Nucleus basalis of Meynert; Organic mental disorders. Neuropsychology; Prosencephalon - cytology; Prosencephalon - metabolism; Prosencephalon - virology; Psychology. Psychoanalysis. Psychiatry; Psychopathology. Psychiatry; Rats; Rats, Inbred F344; Rats, Sprague-Dawley; Trophic activity; Vertebrates: nervous system and sense organs; Neurotrophin; Nucleus basalis of Meynert; Gene delivery; Adeno-associated virus; Dose–response; CERE-110; Trophic activity; Nerve growth factor; Basal forebrain cholinergic neurons; Alzheimer's disease; Cholinergic neuron; Nervous system diseases; Growth; Alzheimer disease; Rodentia; Cerebral disorder; Vertebrata; Mammalia; Treatment; Animal; Central nervous system disease; Degenerative disease; Adeno-associated virus;Alzheimer's disease;Basal forebrain cholinergic neurons;CERE-110;Dose-response;Gene delivery;Nerve growth factor;Neurotrophin;Nucleus basalis of Meynert;Trophic activity

Treatment of degenerating basal forebrain cholinergic neurons with nerve growth factor (NGF) in Alzheimer's disease has long been contemplated, but an effective and safe delivery method has been lacking. Towards achieving this goal, we are currently developing CERE-110, an adeno-associated viru...