2018-043
INVENTORS
Sergio T. Ferreira
Diana Jerusalinsky
William L. Klein*
SHORT DESCRIPTION
A novel gene transfer approach for brain delivery of an antibody for Alzheimer’s disease treatment
BACKGROUND
Alzheimer’s disease, a progressive form of neurodegeneration that causes impairs memory and cognition, currently impacts over 5 million Americans. The number of patients is predicted to triple over the next three decades. The estimated annual cost of Alzheimer’s disease is over $200 billion. Thus, Alzheimer’s disease is in urgent need of novel effective treatments. Accumulation of amyloid-beta oligomers in the brains of patients with Alzheimer’s disease. This finding suggests targeting amyloid-beta oligomer formation and accumulation might modify the progression of Alzheimer’s disease.
ABSTRACT
Scientists at Northwestern have developed a method to prevent and neutralize the neuronal impact of amyloid-beta oligomers, the toxins that accumulate in the Alzheimer’s disease (AD) brain and are thought to affect synapses and cause memory loss. The use of an adeno-associated virus vector to drive neuronal expression of NUsc1, a genetically engineered antibody that binds to amyloid-beta oligomers, produces sustained expression of NUsc1. This gene transfer approach for brain delivery of a specific antibody that binds a specific subset of neurotoxic oligomers results in marked neuroprotection and preservation of learning and memory in animal models of AD.
APPLICATIONS
ADVANTAGES
PUBLICATION
Ding Y, Zhao J, Zhang X, Wang S, Viola KL, Chow FE, Zhang Y, Lippa C, Klein WL, Gong Y. Amyloid Beta Oligomers Target to Extracellular and Intracellular Neuronal Synaptic Proteins in Alzheimer's Disease. Front Neurol. 2019 Nov 1;10:1140.
IP STATUS
US patent application has been filed
Treatment of mice in an Alzheimer’s disease model with AAV-Nusc1 (green bar) significantly improves performance on a memory task compared to Alzheimer’s disease model mice without treatment (red bar)