Drug intelligence / Profile preview

3-pentanone + AAV-hBDNF

Development stage
Unknown
Lead developer
Shiyan Taihe Hospital
Modality
Gene Therapies, Small Molecules
Administration
Intracranial, Inhalation
01

Overview

3-pentanone + AAV-hBDNF is an experimental combination therapy developed by Shiyan Taihe Hospital (Taihe Hospital of Hubei University of Medicine) for the treatment of severe disorders of consciousness (DOC), including vegetative state (VS) and minimal consciousness state (MCS). The therapy utilizes a dual-action approach: a gene therapy component consisting of an adeno-associated virus (AAV) vector carrying the human brain-derived neurotrophic factor (hBDNF) gene, and a small molecule component, 3-pentanone (also known as diethyl ketone). The AAV-hBDNF is typically administered via intracranial injection into targeted brain regions such as the thalamus to promote neuronal survival, synaptic plasticity, and the repair of neural circuits. 3-pentanone is employed as a potent olfactory stimulant to activate the olfactory-thalamic-cortical pathway, aiming to enhance arousal and awareness in patients. This combination seeks to synergistically address the structural and functional deficits underlying persistent consciousness disorders.

Other names
dipentanone + gene therapy virus3-pentanone + AAV-BDNFdiethyl ketone + AAV-hBDNF3-pentanone + adeno-associated virus-human brain-derived neurotrophic factor
02

Targets

OR (Olfactory receptors)NTRK2 (Tropomyosin-related kinase receptor type B)

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