Cedars-sinai Medical Center

Cincinnati Children's Hospital Medical Center

Moise Danielpour

Joshua Breunig

Vivian Hwa

Fataneh Majlessipour

Gi Bum Kim

The present invention describes methods for treating idiopathic short stature. Described herein are also methods of increasing a subject's height. The methods involve administering an effective amount of a pan FGFR inhibitor or a selective FGFR inhibitor to the subject. An example of an FGRF inhibitor used in the methods described herein is erdafitinib.

Use of FGFR inhibitors for treatment of idiopathic short stature

Described herein are donor vectors and systems for use in in vivo dual recombinase-mediated cassette exchange. Also described are animal models for consistent, rigorous, and facile investigation of transgene expression. Further described are methods of screening for therapeutic drugs using these animal models, and methods of treatment.

Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof

Described herein are composition and methods related to targeting the Nf1-Ras-Ets axis, that when perturbed, is identified as playing a role in initiation and maintenance in glioma. A postnatal, mosaic, autochthonous, glioma model that captures the first hours and days of gliomagenesis in more resolution than conventional genetically engineered mouse models of cancer demonstrates that disruption of the Nf1-Ras pathway in the ventricular zone at multiple signaling nodes uniformly results in rapid neural stem cell depletion, progenitor hyperproliferation, and gliogenic lineage restriction. By abolishing Ets subfamily activity, which is upregulated downstream of Ras, there is block of glioma initiation, thereby providing new therapeutic avenues for targeting glioma.

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Los Angeles, CA, United States of America

Moise Danielpour