A rAAV vector is described herein which has an AAVhu68 capsid and at least one expression cassette in the capsid. The at least one expression cassette comprises nucleic acid sequences encoding a functional SMN protein and expression control sequences that direct expression of the SMN sequences in a host cell. Also provided are compositions containing this rAAVhu68.SMN vector and methods of using same for spinal muscular atrophy in a patient.

Provided herein are antibodies that specifically bind Tau and methods of using the same.

The present invention provides methods and compositions relating to an assay for hERG channel protein sensitivity to small molecule pharmacological agents. In one embodiment, the invention includes an engineered hERG channel protein. In another embodiment, the invention includes a method of identifying small molecule pharmacological agents that interfere with repolarization of cardiac cells.

The present invention provides methods and compositions relating to an assay for hERG channel protein sensitivity to small molecule pharmacological agents. In one embodiment, the invention includes an engineered hERG channel protein. In another embodiment, the invention includes a method of identifying small molecule pharmacological agents that interfere with repolarization of cardiac cells.

The present invention discloses a method for assessing the capacity of a substance to treat or prevent hepadnavirus infection. A reporter virus carrying genetic information for a first fragment of a recombinase and a reporter cell expressing a second fragment of the recombinase are used. When the reporter virus infects the reporter cell, the two fragments of the recombinase associate and excise a stop cassette that is flanked by two recombination sites and blocks the expression of a reporter gene. Accordingly, the present invention relates to a method of assessing the capacity of a substance to treat or prevent hepadnavirus infection, a hepadnavirus comprising a nucleic acid encoding a first fragment of a recombinase and a mammalian hepatocyte or hepatoma cell comprising a nucleic acid encoding a second fragment of a recombinase and a nucleic acid comprising a stop cassette flanked by two recombination sites fused to a reporter gene.

Bioluminescent biosensors useful for monitoring and/or quantifying, in vitro or in vivo, activity of the Hippo signaling pathway. The biosensors monitor LATS kinase activity or YAP-TEAD interaction. The biosensors may be used in methods for monitoring and/or quantifying in real-time, in vitro or in vivo, activity of the Hippo signaling pathway, wherein the activity may be LATS kinase activity and/or YAP-TEAD interaction. The biosensors may be provided in kits for monitoring and/or quantifying in real-time, in vitro or in vivo, activity of the Hippo signaling pathway, wherein the activity may be LATS kinase activity and/or YAP-TEAD interaction.

Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor and/or survival factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes a survival factor, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor and/or a different survival factor.

The present invention relates to expression constructs and viral and other vectors for the treatment and/or prevention of chronic pain.

An isolated Methyl-CpG binding domain (MBD) variant may include an MBD core domain having at least 60% sequence homology relative to any one of SEQ ID Nos. 1-45 and comprising at least one amino acid substitution relative to the corresponding wildtype MBD in various positions. The isolated MBD variant or the conjugate may be used for determining the methylation state of cytosine residues and/or oxidation state of 5-methylated cytosine residues in a CpG dinucleotide of interest and its complement in a DNA molecule or for the enrichment of DNA molecules comprising a CpG dinucleotide of interest and its complement. At least one cytosine nucleobase in the CpG dinucleotide may be modified to be 5-methylcytosine (mC), 5-hydroxymethylcytosine (hmC), 5-formylcytosine (fC), or 5-carboxylcytosine (caC).

The present invention relates to a method of producing recombinant binding proteins with binding specificity for a peptide-MHC (pMHC) complex. The invention also relates to recombinant binding proteins comprising one, two or more designed repeat domain(s), preferably designed ankyrin repeat domain(s), with binding specificity for a pMHC complex, and to such binding proteins which further comprise a binding agent having binding specificity for a protein expressed on the surface of an immune cell, preferably a T-cell. In addition, the invention relates to nucleic acids encoding such binding proteins or repeat domains, pharmaceutical compositions comprising such binding proteins or nucleic acids, and the use of such binding proteins, nucleic acids or pharmaceutical compositions in methods for treating or diagnosing diseases, including cancer, infectious diseases and autoimmune diseases.