Disclosed herein are neuronal cell activity reporter systems including a Secreted Neuronal Activity Reporter (SNAR) construct and a control construct. The SNAR construct includes four tandem repeats of a core domain of the Synaptic Activity Response Element (SARE) of Arc/Arg3.1, a polynucleotide comprising the Arc minimal promoter, and a polynucleotide encoding a first secreted reporter protein. The control construct includes a constitutive promoter and a polynucleotide encoding a second secreted reporter protein. Further provided are methods of monitoring neuronal activity in a cell. The methods may include administering to a cell the neuronal cell activity reporter system, contacting with a substrate, and measuring a signal.

Angelman syndrome is a genetic neurological disorder with characteristics including delayed development, intellectual disability, severe speech impairment, and problems with movement and balance. Provided herein are polynucleotides, vectors, polypeptides, cells, compositions, kits and methods to treat Angelman syndrome.

Abstract: Provided herein are methods of producing heparin and heparan sulfate from modified cells, such as modified MST cells and modified basophil neoplastic cells, and compositions comprising heparin and heparan sulfate isolated from modified cells. In some embodiments, methods herein comprise culturing a genetically modified cell line comprising at least one of a mastocytoma cell line and a basophil neoplastic cell line; and isolating the heparin or heparan sulfate from the cell line.

The present invention provides HIV-derived lentivectors which are safe, highly efficient, and very potent for expressing transgenes for human gene therapy, especially, in human hematopoietic progenitor cells as well as in all other blood cell derivatives. The lentiviral vectors comprise a self-inactivating configuration for biosafety and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments such as inherited and acquired lympho-hematological disorders, gene-therapies for cancers especially the hematological cancers, as well as for the study of hematopoiesis via lentivector-mediated modification of human HSCs.

The present invention is directed to methods and compositions that provide therapy for at least one medical condition that directly or indirectly affects cardiac muscle cells (also known as cardiomyocytes) in a mammalian individual, including humans, dogs, cats, horse pigs, and so forth. The medical condition may be of any kind, including a cardiac condition such as heart failure, cardiomyopathy, myocardial infarction, and so forth. The medical condition may have a cardiac condition as its primary symptom or cause or it may be a secondary symptom or cause. The individual may be male or female and may be of any age.

The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.

The present invention relates to a composition for improving memory including a Cyclin Y (CCNY) inhibitor as an active ingredient. More specifically, the present invention relates to a method for improving memory in a subject comprising administering the composition for improving memory to the subject.

The present invention may reveal a CCNY-oriented molecular mechanism with respect to learning and memory, help understand causes of brain diseases associated with memory problems, and ultimately be applied in the treatment and diagnosis of brain memory disorders such as dementia.

Methods of determining subcellular localization of nucleic acids, including RNA and DNA are described. In particular, the invention relates to a method combining proximity-specific labeling with crosslinking of nucleic acids to proteins and sequencing to identify nucleic acids within or near a particular subcellular compartment in vivo.

The invention relates to methods and compositions for treating a neurodegenerative disease. More particularly, the present invention is directed to methods of treatment of neurodegenerative diseases using progranulin and progranulin polypeptides, and methods of treatment of neurodegenerative diseases using effectors, or combinations of effectors, that modify progranulin expression.

Disclosed are means, methods and compositions of matter useful for induction of immunological responses towards tumor endothelial cells. In one embodiment the invention teaches fusion of dendritic cells and cells resembling tumor endothelial cells and administration of such chimeric cells as an immunotherapy for stimulation of tumor endothelial cell destruction. In other embodiments pluripotent stem cells are utilized to generate dendritic cells, wherein said dendritic cells are fused with pluripotent stem cell derived endothelial cells created in a manner to resemble tumor endothelial cells.