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Inhibitor of k-light polypeptide gene enhancer in B cells kinase complex-associated protein (IKBKAP); adrenergic receptor a2 (ADRA2)

A study in neural cells derived from patients with familial dysautonomia (FD) suggests antagonizing ADRA2 could help treat the disease. FD is caused by a mutation in IKBKAP that leads to incorrect splicing and a reduction in IKBKAP protein levels. In neural crest precursors from patient-derived induced pluripotent stem cells, a screen of about 7,000 small molecules identified 43 hits that increased expression of IKBKAP compared with vehicle. Seven of these hits, including a selective ADRA2 antagonist, were further validated in cell culture experiments. Next steps include investigating the mechanism of action for additional screening hits (see Scaling up iPS cells, page 9).

SciBX 6(1); doi:10.1038/scibx.2013.20
Published online Jan. 10, 2013

Unpatented; licensing status not applicable

Lee, G. et al. Nat. Biotechnol.; published online Nov. 25, 2012;
Contact: Lorenz Studer, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center, New York, N.Y.

Contact: Gabsang Lee, The Johns Hopkins University School of Medicine, Baltimore, Md.