The invention relates to synthetic polynucleotides encoding fukutin related protein (FKRP). The invention further relates to nucleic acid constructs comprising the synthetic polynucleotides and methods of using these synthetic polynucleotides to treat dystroglycanopathy disorders.

This disclosure relates to methods for screening therapeutic agents to treat altered function of a mutated target gene (e.g., clinical variant) as well as reagents for use in the same.

Provided herein are compositions comprising light-activated chimeric proteins expressed on plasma membranes and methods of using the same to selectively depolarize excitatory or inhibitory neurons.

The present invention relates to a pharmaceutical composition comprising a compound of formula (I) ##STR00001##
and methods of treating or preventing cell proliferation disorders comprising administering to a subject a therapeutically active amount or a preventive amount of such a compound.

Problem to be Solved: The present invention is intended to provide a polynucleotide encoding the light-chain variable region and the heavy-chain variable region of an anti-dog IgE antibody; and an anti-dog IgE antibody containing these variable regions. Solution: The present invention is DNA encoding a heavy-chain variable region consisting of the amino acid sequence represented by SEQ ID NO: 2 or 6 and DNA encoding a light-chain variable region consisting of the amino acid sequence represented by SEQ ID NO: 4 or 8, and an anti-dog IgE monoclonal antibody which binds to dog IgE, containing these variable regions or a functional fragment thereof which binds to dog IgE.

The present invention relates to the method and use of fluorescent proteins in making transgenic fluorescent ornamental amphibians. The fluorescent ornamental amphibians are used to establish a population of transgenic ornamental amphibians and to provide to the ornamental amphibian industry. Thus, new varieties of ornamental amphibians of different fluorescence colors from a novel source are developed.

The present invention relates to the method and use of reef coral fluorescent proteins in making transgenic red, green and yellow fluorescent zebrafish. Preferably, such fluorescent zebrafish are fertile and used to establish a population of transgenic zebrafish and to provide to the ornamental fish industry for the purpose of marketing. Thus, new varieties of ornamental fish of different fluorescence colors from a novel source are developed.

The present invention encompasses the recognition that identification of alternative means to block RAS oncogenic signaling may be required for developing novel cancer therapies. Among other things, the present invention encompasses the recognition that targeting RAS palmitoylation can achieve effective therapy for RAS-related cancers. Furthermore, the present invention encompasses the recognition that reduction of ZDHHC9 level and/or activity can significantly reduce palmitoylation level of Ras protein. Among other things, the present invention encompasses the recognition that identification of agents that modulate expression and/or activity of ZDHHC9 can reduce palmitoylation level of Ras protein. In some embodiments, the present invention provides methods of treating a subject suffering from cancer by administering ZDHHC9 inhibition therapy.

Methods for treatment and diagnosis of neurological disorders such as autism and autism spectrum disorder are disclosed. Also disclosed are modulators of alternative splicing regulators SRRM4 and/or SRRM3 for treating neurological disorders. Further disclosed are agents that modulate the expression of at least one splice variant for treating neurological disorders. Mouse models of neurological disorders having increased or decreased expression of SRRM4 and/or SRRM3 are also disclosed.

The present invention provides an anti-IGF-I receptor antibody that binds specifically to an IGF-I receptor of a vertebrate and has the proliferation-inducing activity of a vertebrate-derived cell, or a fragment thereof, or derivatives of these.