Novel assay and novel methods of treating hutchinson-gilford progeria syndrome
AssigneeThe Progeria Research Foundation, Inc.
InventorsLeslie B. GORDON, Sarah HAMREN, Robert GOODSON
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Abstract
Provided herein is a newly developed immunoassay that can detect progerin with high sensitivity, but which does not detect wildtype lamin A. Applications of the newly developed immunoassay in novel methods for diagnosing, prognosing, and treating progeria are also described.
Claims22
A method for prognosis of Hutchinson-Gilford progeria syndrome (HGPS) in a subject, comprising:
- providing a sample from a subject;
- quantitating the concentration of progerin in in the sample with a quantitative immunoassay, wherein the quantitative immunoassay detects progerin but does not detect wildtype lamin A protein;
- and
- comparing the concentration of progerin in the sample with an HGPS-positive control, wherein a concentration of progerin in the sample that is below the concentration of progerin in the HGPS-positive control indicates an increased life expectancy in the subject.
(canceled)
A method for determining the efficacy of a treatment for Hutchinson-Gilford progeria syndrome (HGPS), comprising:
- providing a pre-treatment sample from a subject, that is taken prior to administration of a treatment for HGPS;
- determining the concentration of progerin in the pre-treatment sample with a quantitative immunoassay, wherein the quantitative immunoassay detects progerin but does not detect wildtype lamin A protein;
- providing a post-treatment sample from the subject, that is taken during or after administration of a treatment for HGPS;
- determining the concentration of progerin in the post-treatment sample with the quantitative immunoassay;
- and
- comparing the pre-treatment concentration of progerin with the post-treatment concentration of progerin, wherein a significant decrease in progerin concentration in the post-treatment sample indicates that the treatment is effective.
(canceled)
A method for treatment of Hutchinson-Gilford progeria syndrome (HGPS) in a subject, comprising:
- providing a sample from a subject;
- quantitating the concentration of progerin in in the sample with a quantitative immunoassay, wherein the quantitative immunoassay detects progerin but does not detect wildtype lamin A protein;
- comparing the concentration of progerin in the sample with an HGPS-positive control, wherein if the concentration of progerin in the sample indicates that the subject has HGPS;
- and
- if the subject has HGPS, administering to the subject a treatment for HGPS that lowers progerin concentration.
Dependencies are inferred from the claim text.