Abstract: The present disclosure provides methods for treating or ameliorating a myelin associated disease or a mitochondria associated disease that comprising administering to a subject in need thereof a frataxin replacement therapeutic compound, e.g., a fusion protein comprising frataxin.

Abstract: The present disclosure is based, at least in part, on providing a set of markers, also referred to herein as FXN-sensitive lipid markers (or FSLMs), the respective levels of which are positively or negatively correlated to frataxin (FXN) levels in a cell. Therefore, these FSLMs can be used to determine, evaluate, and/or monitor the effectiveness of FXN replacement therapy in a subject.

Abstract: The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents the interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.

Abstract: The present disclosure is based, at least in part, on providing a set of markers, also referred to herein as FXN-sensitive genomic markers (or FSGMs), the respective expression levels of which are positively or negatively correlated to frataxin (FXN) levels in a cell. Therefore, these FSGMs can be used to determine, evaluate, and/or monitor the effectiveness of FXN replacement therapy in a subject.

Abstract: The present disclosure provides a method for treatment of a subject suffering from Leigh Syndrome French Canadian Type (LSFC), the method comprising administering to the subject a therapeutically effective amount of a frataxin (FXN) therapeutic compound.

Abstract: The present disclosure provides methods for determining the amount of FXN or FXN fusion protein in a sample, e.g, a tissue sample, by using mass spectrometry.

Abstract: The present disclosure provides methods for treating or ameliorating a myelin associated disease or a mitochondria associated disease that comprising administering to a subject in need thereof a frataxin replacement therapeutic compound, e.g., a fusion protein comprising frataxin.

Abstract: The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents said interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.

Abstract: The present disclosure provides methods, compositions and kits for measuring activity of a frataxin (FXN) protein, e.g., an FXN fusion protein. The present invention also provides methods for identifying compounds capable of modulating activity of an FXN protein, e.g., an FXN fusion protein.

Abstract: The invention relates to a method of treating a subject who has cancer or a non-malignant tumor, the method comprising administering a therapeutically effective amount of a 18-20 member bi-polycyclic compound to the subject.

Abstract: The present disclosure is based, at least in part, on providing a set of markers, also referred to herein as FXN-sensitive genomic markers (or FSGMs), the respective expression levels of which are positively or negatively correlated to frataxin (FXN) levels in a cell. Therefore, these FSGMs can be used to determine, evaluate, and/or monitor the effectiveness of FXN replacement therapy in a subject.

Abstract: The invention relates to a method of inhibiting loss of muscle mass or muscle function in a subject in need thereof, or a method of treating miofibers ex vivo, the method comprising administering a therapeutically effective amount of a 18-20 member bi-polycyclic compound to the subject or to the miofibers.

Abstract: The invention relates to a method of inhibiting loss of muscle mass or muscle function in a subject in need thereof, or a method of treating miofibers ex vivo, the method comprising administering a therapeutically effective amount of a 18-20 member bi-polycyclic compound to the subject or to the miofibers.

Abstract: The invention relates to a method of treating a subject who has cancer or a non-malignant tumor, the method comprising administering a therapeutically effective amount of a 18-20 member bi-polycyclic compound to the subject.

Abstract: The invention relates to 18-20 member bi-polycyclic compounds, methods of making these compounds, and methods of using them in treating hyperproliferative disorders (e.g., cancer) and non-malignant tumors; promoting muscle formation; inhibiting muscle degeneration or the loss of muscle mass or muscle function; and myofibers ex vivo.

Abstract: The invention relates to 18-20 member bi-polycyclic compounds, methods of making these compounds, and methods of using them in treating hyperproliferative disorders (e.g., cancer) and non-malignant tumors; promoting muscle formation; inhibiting muscle degeneration or the loss of muscle mass or muscle function; and myofibers ex vivo.

Abstract: The invention relates to 18-20 member bi-polycyclic compounds, methods of making these compounds, and methods of using them in treating hyperproliferative disorders (e.g., cancer) and non-malignant tumors; promoting muscle formation; inhibiting muscle degeneration or the loss of muscle mass or muscle function; and myofibers ex vivo.

Abstract: The invention discloses a knockout mouse that is homozygous for a RUNX3 null allele, as a novel model for chronic inflammatory disease. The present invention provides methods of diagnosing and assessing predisposition to chronic inflammatory diseases. The present invention further provides methods for testing agents for effectiveness in treating and/or preventing diseases associated with chronic inflammatory or autoimmune conditions.

Abstract: The invention discloses a knockout mouse that is homozygous for a RUNX3 null allele, as a novel model for chronic inflammatory disease. The present invention provides methods for treating T cell-related inflammatory conditions by cell therapy, gene therapy or antisense for up regulating or down regulating RUNX3. The invention further relates to methods of diagnosing and assessing predisposition to chronic inflammatory diseases. The present invention further provides methods for testing agents for effectiveness in treating and/or preventing diseases associated with chronic inflammatory or autoimmune conditions.