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US 2011/0217715 A1Published application
Gene expression in duchenne muscular dystrophy
AssigneesChildren's Hospital Medical Center, The Regents of the University of California
InventorsBrenda L. Wong, Frank R. Sharp, Lisa Lit
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Abstract
Gene expression in peripheral blood from individuals with Duchenne muscular dystrophy (DMD), compared to control individuals, demonstrated differential gene sets that could be used in a method to diagnose DMD, to evaluate effect of DMD therapy, and/or to evaluate propensity to DMD.
Claims17
A method of monitoring steroid treatment of Duchenne muscular dystrophy (DMD), the method comprising
- determining expression, from blood of a treated individual with DMD administered prednisone, deflazacort, or prednisone and deflazacort, of at least one gene in Table 4;
- comparing expression of the at least one gene from the treated individual with an individual with DMD not administered a steroid,
- evaluating the treated individual's response to prednisone and/or deflazacort administration by determining if the at least one gene in the treated individual is either over-expressed ≧1.5 times or is under-expressed ≧1.5 times compared to expression of the same gene(s) from the individual with DMD not administered a steroid;
- and
- ranking the treated individual's response to treatment based on at least one of
- (a) the extent that over-expression or under-expression exceeds 1.5, or
- (b) the number of genes that are over-expressed or under-expressed,
- where the treated individual monitored has a greater response to treatment when (a) is farther from 1.5, and (b) is farther from 1.
5 more elements
A method of ranking a gene as a target for ameliorating Duchenne muscular dystrophy (DMD), the method comprising
- (a) determining from blood of an individual with DMD administered prednisone and/or deflazacort expression of a gene in Table 4,
- (b) comparing expression of the gene from (a) with expression of the same gene from an individual with DMD not administered prednisone and/or deflazacort, and
- (c) ranking the gene as a target for ameliorating DMD by determining if the gene in (a) is either over-expressed ≧1.5 times or is under-expressed ≧1.5 times compared to the same gene in the individual with DMD not administered prednisone and/or deflazacort, where the gene is a better target the farther (c) is from 1.5.
A method of determining amelioration of prednisone side effects in an individual with Duchenne muscular dystrophy (DMD), the method comprising
- ranking a gene as a target for ameliorating prednisone side effects by
- (a) determining from blood of an individual with DMD administered prednisone expression of at least one gene selected from Table 5,
- (b) comparing the expression in (a) with expression of the same gene(s) from an individual with DMD administered deflazacort, and
- (c) ranking the gene as a target by determining if the at least one gene in (a) is either over-expressed ≧1.5 times or is under-expressed ≧1.5 times compared to the same gene(s) in expression from (b), where the gene is a better target the farther (c) is from 1.5.
1 more element
A method of determining efficacy of treatment of Duchenne muscular dystrophy (DMD), the method comprising
- determining, from blood of an individual with DMD treated with a steroid, expression of at least one gene in Table 4;
- comparing expression of the at least one gene from the treated individual with expression of the same gene in an individual with DMD not treated with a steroid;
- evaluating treatment efficacy by determining if the at least one gene in the treated individual is either over-expressed ≧1.5 times or is under-expressed ≧1.5 times compared to expression of the same gene(s) from individual with DMD not treated with a steroid;
- and
- determining treatment efficacy based on at least one of
- (a) the extent that over-expression or under-expression exceeds 1.5, or
- (b) the number of genes that are over-expressed or under-expressed,
- where the treatment is more efficacious when (a) is farther from 1.5, and (b) is farther from 1.
5 more elements
A kit for monitoring steroid treatment from peripheral blood in an individual with Duchenne's muscular dystrophy (DMD), the kit comprising
- at least one primer, the primer selected from a forward oligonucleotide primer/or and a reverse oligonucleotide primer, the primer annealing to at least one of the genes in Table 4, and
- instructions for using the primer to quantitate the gene(s) in Table 4 in peripheral blood,
- where monitoring is by
- determining expression, from blood of a treated individual with DMD administered prednisone, deflazacort, or prednisone and deflazacort, of at least one gene in Table 4;
- comparing expression of the at least one gene from the treated individual with an individual with DMD not administered a steroid,
- evaluating the treated individual's response to prednisone and/or deflazacort administration by determining if the at least one gene in the treated individual is either over-expressed ≧1.5 times or is under-expressed ≧1.5 times compared to expression of the same gene(s) from the individual with DMD nor administered prednisone and/or deflazacort;
- and
- ranking the treated individual's response to treatment based on at least one of
- (a) the extent that over-expression or under-expression exceeds 1.5, or
- (b) the number of genes that are over-expressed or under-expressed,
- where the treated individual has greater response to treatment when (a) is farther from 1.5, and (b) is farther from 1.
8 more elements
Dependencies are inferred from the claim text.