Abstract: Devices, compositions, and methods are described which provide a tubular nanostructure or a composite tubular nanostructure targeted to a lipid bilayer membrane. The tubular nanostructure includes a hydrophobic surface region flanked by two hydrophilic surface regions. The tubular nanostructure is configured to interact with a lipid bilayer membrane and form a pore in the lipid bilayer membrane. The tubular nanostructure may be targeted by including at least one ligand configured to bind to one or more cognates on the lipid bilayer membrane of a target cell.

Abstract: The present invention addresses the problem of providing a method for obtaining Schwann cells directly (by direct reprogramming) without passing through pluripotent stem cells, such as ES cells or iPS cells. As a means for solving this problem, the present invention provides a method for preparing Schwann cells that includes a step of introducing into somatic cells of a mammal at least one gene selected from the group consisting of SOX10 genes and KROX20 genes, or an expression product thereof.

Abstract: Described are methods and compositions for increasing islet-1 (Isl1) activity (e.g., biological activity) and or expression (e.g., transcription and/or translation) in a biological cell and or in a subject.

Abstract: A method and apparatus are provided for delivering an agent into a cell through the application of nanosecond pulse electric fields (“nsPEF's”). The method includes circuitry for delivery of an agent into a cell via known methods followed by the application of nanosecond pulse electric fields to said cell in order to facilitate entry of the agent into the nucleus of the cell. In a preferred embodiment, the present invention is directed to a method of enhancing gene expression in a cell comprising the application of nanosecond pulse electric fields to said cell. An apparatus for generating long and short pulses according to the present invention is also provided. The apparatus includes a pulse generator capable of producing a first pulse having a long duration and low voltage amplitude and a second pulse having a short duration and high voltage amplitude.

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

Abstract: Provided herein are bi-specific chimeric antigen receptors (CARs), such as those specific for CD138 and BCMA. Use of the CARs in immune cells (e.g., T cells), compositions, and methods are also contemplated.

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

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

Abstract: The present invention relates to novel therapeutic ways for treating Wolfram Syndrome (WS) by targeting the neuronal calcium sensor 1 (NCS1). The present inventors have demonstrated that WFS1, which loss of function is responsible of the Wolfram Syndrome, forms a complex with the neuronal calcium sensor 1 (NCS1). The inventors have further demonstrated that WFS1 associates with NCS1 to prevent its degradation by the proteasome and that NCS1 regulates VDAC expression and mitochondrial respiratory chain. Thus, present invention provides an agonist of NCS1 for use in the treatment of WS. Such an agonist is e.g. a NCS1-encoding polynucleotide, an inhibitor of the proteasome or of calpains. The inventors have further shown that overexpression of NCS1 in WS cells allows increasing complex II driven respiration. The present invention further relates to a method for predicting the severity of WS by measuring the NCS1 level in a sample obtained from a patient.

Abstract: Plasmonics-active nanoprobes are provided for detection of target biomolecules including nucleic acids, proteins, and small molecules. The nucleic acids that can be detected include RNA, DNA, mRNA, microRNA, and small nucleotide polymorphisms (SNPs). The nanoproprobes can be used in vito in sensitive detection methods for diagnosis of diseases and disorders including cancer. Multiplexing can be performed using the nanoprobes such that multiple targets can be detected simultaneously in a single sample. The methods of use of the nanoprobes include detection by a visible color change. The nanoprobes can be used in vivo for treatment of undesirable cells in a subject.

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

Abstract: The present invention relates to a novel Hepatitis B virus (HBV) and/or Hepatitis D virus (HDV) receptor and its use for the development of cells, cell lines and non-human animals that are susceptible to HBV and/or HDV infection and can be used for immunological studies and/or for the screening of drugs, post-entry restriction factors and host dependency factors. It further relates to the use of the receptor for the identification of compounds useful in the treatment of HBV and/or HDV infection.

Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic cell.

Abstract: The present invention provides novel nucleic acid sequence compositions and methods relating to minimal human PAX6 promoters. The invention is based in part on the surprising discovery that certain minimal PAX6 promoter elements are capable of expressing in specific cell types in cells eye.

Abstract: A bis-alkoxyl amide alkyl cationic peptide lipid has a chemical structure as below: wherein the bis-alkoxyl amide alkyl cationic peptide lipid is dispersed in water to obtain the cationic peptide liposome which are high in stability and uniform in dispersion and have about 120 nm of average grain diameter and Zeta electric potential between 30 and 50 mV. The liposome can effectively compress the plasmids DNA and siRNA, can efficient transfection both in-vitro and in-vivo, and almost does not have toxicity to cells and mice, so that the liposome can be widely applied in gene delivery as a gene vector.

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

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

Abstract: To provide a base sequence for protein expression that can increase the yield of protein such as diastatic enzyme by further activating a promoter of a particular gene. A base sequence 1 for protein expression includes: a gene 3 encoding protein 2; a promoter 4 of the gene 3, the promoter being linked upstream of the gene 3; and a cis element 5 whose activity is improved by an artificial transcription factor 6, the cis element being linked further upstream of the promoter 4. The cis element 5 is represented by SEQ ID NO: 1.

Abstract: The present invention relates to compositions and methods for creation of vector nucleic acid sequences (e.g., retroviral nucleic acid sequences) that comprise two or more exogenous nucleic acid sequences that encode highly homologous (e.g., identical) polypeptide sequences, yet wherein at least one of the exogenous nucleic acid sequences has been mutated using degenerate codons for purpose of reducing homology between the two or more exogenous nucleic acid sequences while maintaining the encoded polypeptide sequence. Preferred nucleic acid sequences include those encoding multi-chimeric immune receptor (CIR) genes. Specific nucleic acid sequences of such CIR genes are also disclosed.

Abstract: The present invention refers to a device, comprising a hollow body having at least one open end comprising at least one solid matrix binding, adsorbing, absorbing, chelating or retaining compounds which are not desired in a sample and preferably at least one barrier which is non-permeable for liquids and solids under ambience conditions, however, becomes liquid-permeable by applying an external force to the barrier, the use of such a device for isolating or purifying a biomolecule from a sample, a method for preparation of the device and a method for isolation or purification of any biomolecule using said device.