Abstract: In alternative embodiments, the invention provides nucleic acid sequences that are genetic polymorphic variations of the human TMEM216 gene, and TMEM216 polypeptide encoded by these variant alleles. In alternative embodiments, the invention provides methods of determining or predicting a predisposition to, or the presence of, a ciliopathy (or any genetic disorder of a cellular cilia or cilia anchoring structure, basal body or ciliary function) in an individual, such as a Joubert Syndrome (JS), a Joubert Syndrome Related Disorder (JSRD) or a Meckel Syndrome (MKS). In alternative embodiments, the invention provides compositions and methods for the identification of genetic polymorphic variations in the human TMEM216 gene, and methods of using the identified genetic polymorphisms and the proteins they encode, e.g., to screen for compounds that can modulate the human TMEM216 gene product, and possibly treat JS, JSRD or MKS.

Abstract: The present invention relates to cell specific therapeutic modality by using a region of the PLAP Promoter. The invention further relates to specific expression of therapeutically PLAP useful sequences for specific transcriptional activation of this gene. The invention also relates to the PLAP region which may be used alone or in combination with other regions like enhancer sequences that augment cell or tumour specific gene expression.

Abstract: The present invention relates to a pharmaceutical composition comprising a compound of formula (I) 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.

Abstract: The present invention concerns methods and compositions for the treatment of cancer and cancer cells using intravascular administration of a vaccinia virus. In some embodiments, methods and compositions involve a replicative vaccinia virus that encodes GM-CSF.

Abstract: The present invention relates to a composition for stimulating hair growth or preventing hair loss, which contains a conditioned medium or extract of neural stem cells (NSCs) isolated from the ventricular zone of the human brain, and to a preparation method thereof. The conditioned medium or extract of neural stem cells according to the present invention contains various growth factors and cytokines, and thus has an excellent effect on the stimulation of hair growth. Thus, it is useful for hair growth stimulation and hair loss prevention.

Abstract: Disclosed herein are methods of detecting influenza virus in a sample from a subject. In some embodiments, the disclosed methods include contacting a sample with at least one primer 10-40 nucleotides in length wherein the at least one primer is capable of hybridizing to an influenza virus polymerase basic protein 1 (PB1) nucleic acid at least 70% identical to the nucleic acid sequence set forth as any one of SEQ ID NOs: 1-3, amplifying the PB1 nucleic acid or a portion thereof to produce an amplified PB1 nucleic acid, and detecting the amplified PB1 nucleic acid, wherein presence of the amplified PB1 nucleic acid indicates presence of influenza virus in the sample from the subject. In some examples, the primers comprise or consist of the nucleic acid sequence set forth as one of SEQ ID NO: 4, SEQ ID NO: 5, SEQ ID NO: 9, or SEQ ID NO: 10.

Abstract: The present invention generally relates to microcolumn affinity chromatography devices, systems that include the microcolumn affinity chromatography devices of the present disclosure, methods of using the devices and the systems of the present disclosure, and methods of making the devices and the systems of the present disclosure. In certain embodiments, the microcolumn affinity chromatography device is suitable for conducting affinity chromatography in multiple microcolumns in parallel and/or in series.

Abstract: The present invention provides for nucleic acids improved for the expression of interleukin-15 (IL-15) in mammalian cells. The invention further provides for methods of expressing IL-15 in mammalian cells by transfecting the cell with a nucleic acid sequence encoding an improved IL-15 sequence. The present invention further provides expression vectors, and IL-15 and IL-15 receptor alpha combinations (nucleic acid and protein) that increase IL-15 stability and potency in vitro and in vivo. The present methods are useful for the increased bioavailability and biological effects of IL-15 after DNA, RNA or protein administration in a subject (e.g. a mammal, a human).

Abstract: The preset invention relates to a promoter to target a fluorescent protein to the muscles of fish, such as A. nigrofasciatus, for ornamental purposes, which is a Mlc3 (myosin, light polypeptide 3, skeletal muscle) promoter. The Mlc3 promoter has the nucleotides of tilapia (Oreochromis niloticus) myosin light chain 3 (Mlc3) promoter region, which is potential to be a tilapia Mlc3 promoter to enhance protein expression in muscle of fish, particularly for the generation of ornamental fish.

Abstract: The present invention provides attenuated Toxoplasma gondii mutants for use as vaccines in the prevention or treatment of cancer.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted integration of anti-HIV transgenes into the genome of a cell for the treatment and/or prevention of HIV.

Abstract: The invention concerns the use of pyridinium furosemide or one of the derivatives, analogs, salts, metabolites, or prodrugs thereof in the preparation of a chemical model of a neurodegenerative disease, preferably Parkinson's disease. The invention also concerns the corresponding chemical model and the uses of same, in particular in screening tests for identifying drug candidates.

Abstract: The present invention relates to compounds for use in modulating the toll-like receptor 4 (TLR4) signaling pathway, as well as to a pharmaceutical composition comprising said compounds.

Abstract: A serum-stable mixture of lipids capable of encapsulating an active agent to form a liposome, said mixture comprising phosphatidylcholine and phosphatidylethanolamine in a ratio in the range of about 0.5 to about 8. The mixture may also include pH sensitive anionic and cationic amphiphiles, such that the mixture is amphoteric, being negatively charged or neutral at pH 7.4 and positively charged at pH 4. Amphoteric liposomes comprising such a mixture may be used for encapsulating nucleic acid therapeutics, such as oligonucleotides and DNA plasmids. The drug/lipid ratio may be adjusted to target the liposomes to particular organs or other sites in the body.

Abstract: Aptamers consisting of a single stranded nucleic acids having 100 nucleotides or less that specifically binds to tumor initiating cancer cells are described. The aptamers can be identified by screening a large pool of randomly generated aptamers to obtain a discrete set of aptamers that specifically bind to tumor initiating cancer cells, such as those found in brain cancer or glioblastoma. The aptamers can also be linked or complexed with anticancer agents or imaging agents for use in therapy or diagnosis.

Abstract: A method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first, second or third vector, wherein the first vector comprises a promoter enhancer, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase and an albumin 3?UTR and lacks an HGH intron, wherein the second vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site and an albumin 3?UTR and lacks a promoter enhancer, wherein the third vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site, an albumin 3?UTR and a promoter enhancer and observing the patient's insulin levels, wherein the patient's insulin levels are controlled.

Abstract: The present invention provides for nucleic acids improved for the expression of interleukin-15 (IL-15) in mammalian cells. The invention further provides for methods of expressing IL-15 in mammalian cells by transfecting the cell with a nucleic acid sequence encoding an improved IL-15 sequence. The present invention further provides expression vectors, and IL-15 and IL-15 receptor alpha combinations (nucleic acid and protein) that increase IL-15 stability and potency in vitro and in vivo. The present methods are useful for the increased bioavailability and biological effects of IL-15 after DNA, RNA or protein administration in a subject (e.g. a mammal, a human).

Abstract: Disclosed herein are methods and compositions for modulating activity of CXCR4 genes, for example using zinc finger transcription factors (ZF-TFs) or zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZF-TFs or ZFNs, vectors comprising polynucleotides encoding ZF-TFs or ZFNs and cells comprising polynucleotides encoding ZF-TFs or ZFNs and/or cells comprising ZF-TF or ZFNs are also provided.

Abstract: A method of tracking cell identity across analytical platforms uses stochastic barcoding (SB). SB uses a randomly generated code based on one or more of the number, color and position of beads encapsulated together with a set of cells of interest. SB use is demonstrated in an application where cells are transferred from a microwell array into a microtiter plate while keeping their identity, and obtained an average identification accuracy of 96% for transfer of 100 blocks. Model scaling of the method up to 1000 blocks demonstrated that SB is able to achieve approximately 90% accuracy.

Abstract: The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.