Abstract: Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSGCA using a systemic route of administration and at a dose of about 1.0×1012 vg/kg to about 5.0×1015 vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.

Abstract: Disclosed herein are methods for preventing or treating non-arteritic anterior ischemic optic neuropathy in a subject by administering to the subject a nucleic acid molecule comprising a nucleic acid sequence encoding OCT4, a nucleic acid sequence encoding SOX2, and a nucleic acid sequence encoding KLF4.

Abstract: The present disclosure provides methods of treating lysosomal storage disorders, e.g., Fabry disease, Gaucher disease, Farber disease, and Pompe disease. The method comprises producing vector-transduced T-Rapa cells that express a transgene of interest and administering the cells to a patient in need thereof. The T-Rapa cells may be transduced with a dual promoter lentivirus vector.

Abstract: Described herein are codon optimized nucleic acid sequences encoding gap junction protein beta 2 (GJB2), and associated genetic elements, for use in recombinant adeno-associated virus (rAAV)-based gene therapy. The rAAV vectors through which GJB2 is packaged can be used for targeted delivery to patients suffering from genetic hearing loss, including patients with autosomal mutations, recessive or dominant, in the GJB2 gene.

Abstract: A treatment of various eye conditions relating to eye enlargement is disclosed. The conditions can be treated with by inhibiting an upstream protein within the biological pathway or by increasing the expression of a downstream receptor within the same pathway. Inhibition of the upstream protein, sterol regulatory element binding protein (SREBP), has been achieved using small molecule inhibitors or nucleic acid in viral vector and increased expression of the downstream protein, bone morphogenetic protein (BMP), has been achieved by nucleic acid in viral vector. Also disclosed are the small molecule, nucleic acid and the viral vector as well as methods of treating ocular diseases.

Abstract: Described herein are compositions comprising a recombinant retroviral vector for delivering a therapeutic comprising a nucleic acid construct comprising a polynucleotide sequence encoding an interleukin or a subunit thereof. Also described herein are methods of using the composition comprising a recombinant retroviral vector described herein for delivering a therapeutic comprising a nucleic acid construct comprising a polynucleotide sequence encoding an interleukin or a subunit thereof to a subject.

Abstract: The invention relates to sulfamidase (SGSH) and SGSH variants. SGSH and SGSH variants can be delivered by way of a recombinant adeno-associated virus (rAAV) particle to a mammal's central nervous system (CNS) to transduce CNS cells that contact cerebrospinal fluid (CSF). Target mammals for SGSH and SGSH variant administration include mammals with a deficiency or defect in SGSH expression or function.

Abstract: Provided herein are genetically modified pigs, porcine organs, tissue, and cells having a reduced propensity to cause a rejection response in a human subject following xenotransplantation. In particular, provided herein are genetically modified pigs lacking nonGal xenoantigens, and porcine cells, tissues, and organs obtained from such genetically modified pigs that are suitable for transplantation into a human. Also provided herein are methods of improving a rejection related symptom in a human subject.

Abstract: The invention relates to retrieving or modifying target nucleic acids, such as host cell chromosomal DNA, by homologous recombination with vectors that have been cut to provide recombinogenic nucleic acid strands in situ. The methods described herein can be used to modify a target nucleic acid in vitro or in any prokaryotic or eukaryotic cell using homologous recombination.

Abstract: The present invention relates to the field of oncolytic viruses and in particular to a recombinant rhabdovirus, such as vesicular stomatitis virus encoding in its genome for a CD80 extracellular domain Fc-fusion protein. The invention is further directed to the use of the recombinant virus in the treatment of cancer, and also to methods for producing such viruses.

Abstract: The invention is to articles of extracellular matrix. The articles comprise one or more sheets of mammalian extracellular matrix laminated together. A single sheet can be folded over and laminated on 3 sides. Two or more sheets can be laminated to each other at their edges. The sheets can further encase a composition comprising a cell or cells, such as for example, a stem cell. A single sheet can be folded over to encase a composition, or rolled to encase a composition with lamination at either end of the roll, for example. The invention also includes methods of using these articles to regenerate tissue at tissue defects, or heal wounds in damaged tissue.

Abstract: In some aspects, cardiac-specific expression cassettes are provided herein. In some aspects, provided herein is an expression cassette comprising a polynucleotide sequence encoding a gene product for therapy of a heart disease, wherein the polynucleotide sequence is operably linked to promoter (e.g., a cardiac-specific promoter), and optionally an enhancer (e.g., a cardiac-specific enhancer). In some aspects, the disclosure provides recombinant adeno-associated virus (rAAV) virions, comprising a capsid protein and a viral genome comprising an expression cassette comprising a polynucleotide sequence encoding a therapeutic gene product, e.g., dwarf open reading frame (DWORF) polypeptide, operably linked to a promoter, the expression cassette flanked by inverted terminal repeats. The disclosure further provides pharmaceutical compositions and methods of treating or preventing heart disease.

Abstract: The present invention provides a nucleic acid aptamer specifically recognizing ?-lactoglobulin and use thereof. The nucleic acid aptamer has a sequence as shown in SEQ ID NO:1, a sequence having 60% or higher homology to the sequence as shown in SEQ ID NO:1 and specifically recognizing ?-lactoglobulin, or a sequence derived from the sequence as shown in SEQ ID NO:1 and specifically recognizing ?-lactoglobulin. The nucleic acid aptamer specifically binds to the allergen ?-lactoglobulin in cow milk and dairy products, thereby providing a new tool for the high-sensitivity and low-cost detection of the allergen ?-lactoglobulin.

Abstract: A method of modulating some or all copies of a gene in a cell is provided including introducing into a cell one or more ribonucleic acid (RNA) sequences that comprise a portion that is complementary to all or a portion of each of the one or more target nucleic acid sequences, and a nucleic acid sequence that encodes a Cas protein and maintaining the cells under conditions in which the Cas protein is expressed and the Cas protein binds and modulates the one or more target nucleic acid sequences in the cell.

Abstract: The present disclosure relates to a preparation of CD140a/PDGFR? positive cells that comprises oligodendrocyte progenitor cells co-expressing OLIG2 and CD140a/PDGFR?. The preparation of cells is derived from pluripotent cells that were derived from skin cells, fibroblasts, umbilical cord blood, peripheral blood, bone marrow, or other somatic cells. The cell preparation has an in vivo myelination efficiency that is equal to or greater than the in vivo myelination efficiency of a preparation of A2B5+/PSA-NCAM? sorted fetal human tissue derived oligodendrocyte progenitor cells. Methods of making, isolating and using the disclosed cell preparation are also described.

Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.

Abstract: The invention relates to synthetic liver-specific promoters and expression constructs for producing polypeptides and functional nucleic acids in the liver of a subject. The invention further relates to Factor VIII proteins containing modifications in the amino acid sequence of the Factor VIII protein, as well as nucleic acid constructs encoding the Factor VIII proteins and methods of using these compositions to treat a bleeding disorder.

Abstract: A method for producing eyefield progenitor cells, including: (a) obtaining a starting population comprising human pluripotent stem cells (hPSCs) that are dissociated to essentially single cells; (b) culturing said hPSCs to a contact-inhibited monolayer; (c) contacting said hPSC monolayer in a primary differentiation medium to generate a homogeneous, contact-inhibited monolayer of anterior neuroectodermal cells (ANECs); (d) dissociating said homogeneous ANECs from (c) into essentially single cells; (e) forming dissociated ANECs into size-controlled and homogeneous 3D aggregates (ANEBs), wherein the ANEBs are 3D aggregates of anterior neuroectodermal cells that are distinct from embryoid bodies; and (f) culturing said ANEBs in a primary differentiation medium in suspension to further differentiate them to Eyefield Progenitor Cells (EFPCs).

Abstract: The disclosure provides a non-human transgenic animal having at least one CREBBP gene locus into which a mutation has been introduced, wherein the mutated CREBBP gene encodes a mutant CREBBP consisting of an amino acid sequence having at least 90% identity with the amino acid sequence of SEQ ID NO: 5. The disclosure also provides a method of producing the transgenic animal comprising introducing a mutation into at least one CREBBP gene locus of a non-human animal. The disclosure further provides use of the transgenic animal as a model of Rubinstein-Taybi syndrome or advanced sleep phase syndrome.

Abstract: A novel method of inducing neural progenitor cells, oligodendrocyte progenitor cells, oligodendrocytes from human iPSCs at an unprecedented efficiency and functionality. The core of the invention is the use of experimentally discovered transcription factors at multiple critical differentiation decision points along a pluripotent to ectoderm, neural ectoderm to NPCs to OPCs to oligodendrocytes pathway in a previously unknown manner.