Methods and compositions for the identification and treatment of individuals having or likely to develop short stature
AssigneeChildren's Hospital Medical Center
InventorsAndrew Nahum Dauber, Vivian Hwa
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Abstract
The instant disclosure relates to methods useful for the treatment or prevention of progressive growth failure in a subject in need thereof. The method may include the step of administering a PAPPA2 gene product to the subject. Also disclosed is the identification of a novel gene mutation that may be used as a marker to identify subjects particularly suited for such treatment. Compositions containing a therapeutically effective amount of PAPPA2 protein and a pharmaceutically acceptable carrier are also disclosed.
Claims28
A method of treating progressive growth failure in a subject in need thereof, comprising the step of administering a PAPPA2 gene product said subject.
A method of determining whether an individual is a candidate for treatment of short stature via administration of a functional PAPPA2 gene product or a recombinant vector containing a nucleic acid sequence that encodes said functional PAPPA2 gene product, comprising the step of determining the presence of a PAPPA2 functional mutation, wherein, if a functional mutation of PAPPA2 is identified, said individual is a candidate for IGF-1 treatment.
A composition comprising a therapeutically effective amount of PAPPA2 protein and a pharmaceutically acceptable carrier.
A composition consisting essentially of a therapeutically effective amount of PAPPA2 protein and a pharmaceutically acceptable carrier.
A composition consisting essentially of a therapeutically effective amount of PAPPA2 protein, a therapeutically effective amount of growth hormone, and a pharmaceutically acceptable carrier
Dependencies are inferred from the claim text.