Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Peptide containing doai
Reexamination Certificate
2004-10-01
2010-06-15
Vogel, Nancy (Department: 1636)
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Peptide containing doai
C435S441000, C435S440000, C536S023100, C536S024300
Reexamination Certificate
active
07737110
ABSTRACT:
This invention relates to methods for altering the splicing of mRNA in cells. In particular, this invention also relates to methods for increasing the ratio of wild type to misspliced forms of mRNA and corresponding encoded proteins in cells possessing a mutant gene encoding either the i) misspliced mRNA corresponding to the mutant protein or ii) a component in the splicing machinery responsible for processing the misspliced mRNA. In addition, this invention relates to treating individuals having a disorder associated with a misspliced mRNA, such as Familial Dysautonomia or Neurofibromatosis 1, by administering to such an individual a cytokinin such as kinetin.
REFERENCES:
Hims, et al. Therapeutic potential and mechanism of kinetin as a treatment for the human splicing disease familial dysautonomia, J. Mol. Med. 85:149-161, 2007.
Slaugenhaupt SA, Gusella JF. Familial dysautonomia.Curr Opin Genet Dev. Jun. 2002;12(3):307-11.
Pavlica et al., “Effects of Benzyladenine on Prokaryotic and Eukaryotic Cells”,Mutation Research, Apr. 1992, vol. 281, No. 4, pp. 277-282.
Cuajungco et al., Tissue-specific Reduction in Splicing Efficiency of IKBKAP due to the Major Mutation Associated with Familial Dysautonomia,Am. J. Hum. Genet., Mar. 2003, vol. 72, No. 3, pp. 749-758.
PCT Search Report dated Feb. 26, 2007.
Anderson et al., “Tocotrienols Induce IKBKAP Expression: A Possible Therapy for Familial Dysautonomia”, Biochem. and Biophys. Res. Comm., Jun. 2003, vol. 36, pp. 303-309.
Anderson et al., “EGCG Corrects Aberrant Splicing of IKAP mRNA in Cells From Patients With Familial Dysautonomia”, Biochem. and Biophys. Res. Comm., Oct. 2003, vol. 310, pp. 627-633.
Slaugenhaupt et al., “Rescue of a Human mRNA Splicing Defect by the Plant Cytokinin Kinetin”, Human Molecular Genetics, Feb. 2004, vol. 13, No. 4, pp. 429-436.
Gold-von Simon et al., “Kinetin in Familial Dysautonomia Carriers: Implications for a New Therapeutic Strategy Targeting mRNA Splicing,” Pediatric Research, 65(3), 341-346 (Mar. 2009)(electronically published ahead of print on Nov. 19, 2008).
Lee et al., “Modeling Pathogenesis and Treatment of Familial Dysautonomia Using Patient-Specific iPSCs,” Nature, 461, pp. 402-408, Sep. 17, 2009.
Gusella James F.
Slaugenhaupt Susan A.
Brivanlou Margaret B.
Hibbert Catherine
King & Spalding LLP
Sonnenfeld Kenneth H.
The General Hospital Corporation
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