Abstract: The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.

Abstract: Methods and uses for diagnosing and treating anxiety disorders are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: This invention relates to methods of treating and ameliorating congenital and neonatal hyperinsulinism and post-prandial hypoglycemia, comprising the step of administering an antagonist of the Glucagon-Like Peptide-1 (GLP-1) receptor, e.g. a GLP-1 fragment or analogue thereof.

Abstract: The present disclosure is directed to antibodies binding to Glypican 2 and methods of using such antibodies to treat cancers that express or overexpress the Glypican 2 antigen.

Abstract: Compositions and methods for the diagnosis and treatment of Covid 19 are disclosed.

Abstract: Methods for diagnosing and treating conduct disorder are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.

Abstract: Compositions for the identification of patients at risk for the development of latent autoimmune diabetes, and methods of use thereof, for the diagnosis and treatment of the same.

Abstract: A biometric sensor device system including a biometric sensor device, and a prediction computer. The biometric sensor device includes at least one camera, and a biometric sensor device processor configured to record a time synchronized communicative interaction between participants, by controlling at least one camera to record the participants over a time period, and transfer the recorded communicative interaction to the prediction computer. The prediction computer including a prediction computer processor configured to extract, from the recorded communicative interaction, a physical characteristic of each of the participants over the time period, compare the physical characteristic of at least one of the participants with the physical characteristic of at least another one of the participants over the time period, and classify or score at least one of the participants according to a predetermined classification or dimensional scoring scheme based on the comparison.

Abstract: Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use.

Abstract: Provided herein are engineered red blood cells expressing rare blood antigen group profiles, and methods of making use the same, are described. Also provided are recombinant reagent red blood cells that express or lack the expression of at least one protein (e.g., a blood group antigen) on its surface and uses thereof.

Abstract: Provided are macromolecular prodrugs in which camptothecin analogs are covalently bonded to polymers via ester bonds that are labile under physiological conditions. Also provided are methods of treating cancer, especially neuroblastoma with the macromolecular prodrugs.

Abstract: Compositions and methods for the detection and treatment of ADHD are provided.

Abstract: The invention relates to compositions and methods as part of a pharmacotherapeutic strategy that targets the endochondral ossification process in a pharmacologically selective and site-specific manner. A variety of orthopedic pathologies are caused by or associated with generalized or local dysregulation of endochondral ossification, for example trauma to the bone growth plate or diaphysis can cause a serious imbalance in bone growth, leading to progressive deformity that today can only be treated surgically. Dysregulated endochondral ossification is also behind heterotopic ossification, which arises in soft tissues and causes pain, decrease in mobility and other clinical problems. The invention therefore provides RARy agonist and antagonist nanoparticle compositions for treating abnormal endochondral ossification and bone growth that can deliver robust local therapeutic control over a particular long bone's growth with a long-lasting effect.

Abstract: The present disclosure is directed to antibodies binding to Glypican 2 and methods of using such antibodies to treat cancers that express or overexpress the Glypican 2 antigen.

Abstract: Compositions and methods for the treatment and diagnosis of eosinophilic esophagitis are disclosed.

Abstract: Methods and uses for diagnosing and treating anxiety disorders are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: Methods for diagnosing and treating conduct disorder are encompassed, wherein diagnosis and treatment may be based upon an assessment of genetic alterations in metabotropic glutamate receptor (mGluR) network genes and wherein treatment is with nonspecific activators of mGluRs such as fasoracetam.

Abstract: In accordance with the present invention, a method for increasing the yield of rLV vector particles comprising a trans gene encoding a therapeutic protein or fragment thereof is disclosed. In one approach, cells are transfected with plasmids encoding the necessary components for rLV production using a calcium chloride transfection mix at pH 7.1 wherein the calcium chloride and plasmids form a complex which is added to the cells at a constant speed. The cells are then incubated for a suitable time period wherein virus particle media is collected at least twice during the incubation period and stored in a cold storage unit, thereby reducing virus inactivation.

Abstract: Provided herein are targeting peptides and vectors containing a sequence that encodes the targeting peptides that deliver agents to specific substructures in the brain. Provided herein are viral vectors each comprising a modified capsid, wherein the modified capsid comprises at least one amino acid sequence that targets the viral vector to a distinct brain structure.