Companion diagnostic to monitor the effects of gene therapy
OwnerAstrid VALLES-SANCHEZ, Pavlina Stefanova KONSTANTINOVAinventors
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Abstract
The invention relates to the field of gene therapy. In addition the invention relates to the field of interfering RNA and/or microRNA (miRNA). In particular the invention relates to gene therapy involving such miRNA's and more in particular to diagnostic tools to be implemented in the treatment of disease, when such treatment is carried out by delivery of miRNA's to a patient, i.e. to cells of a patient. The present invention uses the content of extracellular vesicles that are found in body fluids to determine the presence of miRNA's or the like expressed through the introduction of a gene delivery vehicle is in a host.
Claims17
A method for determining expression of at least one artificial miRNA in the brain, comprising:
- (a) obtaining a sample of cerebrospinal fluid (CSF) or blood (serum/plasma) from a patient having been treated with artificial miRNA, and
- (b) determining abundance of the artificial miRNA in extracellular vesicles containing at least part of the miRNA in the CSF or blood (serum/plasma) sample.
Dependencies are inferred from the claim text.