Abstract: This disclosure provides compositions and methods for improving immunotherapy, specifically against diseases like HIV or lymphoma that manifest within B cell follicles.

Abstract: The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.

Abstract: There is provided a method for efficient differentiation induction from human pluripotent stem cells into hypothalamic neurons. Also, provided is a method for constructing, from human pluripotent stem cells, a cellular structure in which hypothalamic tissue and pituitary tissue are integrated. A cellular structure including hypothalamic tissue is obtained by a method including the steps of: culturing an aggregate of human pluripotent stem cells in suspension in a medium containing a low concentration of a bone morphogenetic protein signal transduction pathway activating substance and a low concentration of a substance acting on the Shh signaling pathway; and further culturing the cell aggregate obtained in the step in suspension in a medium containing a low concentration of a substance acting on the Shh signaling pathway.

Abstract: The present invention is to provide a 3D cartilage organoid block prepared by differentiating mesenchymal stem cells into 3D spheroid cartilage tissues, a basic unit for the 3D cartilage spheroid block. The inventors found that both the amount of GAG matrix and the expression of the collagen type2 increased. Therefore, the method of this invention provides clinically applicable cartilage tissues by effectively enhancing the function of the cartilage differentiation constructs according to 2D culture. The 3D cartilage organoid block can be usefully applied to the area, such as, articular cartilage regeneration and plastic surgery, where cartilage tissues restoration is required.

Abstract: A genetic construct comprises a promoter operably linked to a first coding sequence, which encodes tyrosine hydroxylase (TH), and a second coding sequence, which encodes GTP cyclohydrolase 1 (GCH1), wherein the second coding sequence is 3? to the first coding sequence, and the first and second coding sequences are part of a single operon. The genetic construct does not encode aromatic amino acid decarboxylase (AADC).

Abstract: Provided herein are lentiviral vectors comprising a mutated, heterologous envelope protein, a targeting protein, and at least one transgene for delivery to and expression by a cell characterized by the targeting protein. Also provided are methods and materials for producing the lentiviral vectors described herein, methods for transducing target cells, and cells transduced by lentiviral vectors according to the present disclosure.

Abstract: Some embodiments of the present disclosure relate to one or more compositions that upregulate the production of one or more sequences of mRNA. The sequences of mRNA may encode for translation of a target biomolecule, thereby causing an increase in bioavailability of the target biomolecule within a subject that is administered the one or more compositions. In some embodiments of the present disclosure, the target biomolecule is a protein, such as p53.

Abstract: This invention relates to a truncated dysferlin nucleic acid and protein, vectors (e.g., adeno-associated virus vectors) comprising the nucleic acid and methods of using the same for delivery of dysferlin to a cell or a subject and treating dysferlinopathy.

Abstract: Entomopathogenic nematode Heterorhabditis bacteriophora having an enhanced longevity, comprising a first locus comprising a single nucleotide polymorphism at position 75 of the nucleotide sequence SC00004647 as depicted in SEQ ID NO: 5, in which C is substituted by T; and/or a second locus comprising a single nucleotide polymorphism at position 54 of the nucleotide sequence SC00006203 as depicted in SEQ ID NO: 7, in which C is substituted by T.

Abstract: Disclosed herein are high transducing replication defective herpes simplex virus (HSV) vectors of McKrae strain.

Abstract: This invention relates to polynucleotides comprising a nucleotide sequence encoding a PPT1 polypeptide or a fragment thereof, vectors (viral or non-viral vectors) comprising the same, and methods of using the same for delivery of the open reading frame to a cell or a subject and to treat infantile neuronal lipofuscinosis (infantile Batten disease). The polynucleotides comprise an optimized CLN1 open reading frame.

Abstract: Described herein are systems, methods, and compositions capable of targeting nucleic acids. Describe in certain exemplary embodiments herein are a class of small Cas proteins (Type II-D Cas proteins) and systems thereof. Also described in certain exemplary embodiments herein are methods of modifying target sequences using the class of small Cas proteins (Type II-D Cas proteins) and systems thereof described herein.

Abstract: An objective of the present invention is to provide an improved negative-strand RNA viral vector and a use thereof, the negative-strand RNA viral vector exhibiting transient high expression of genes loaded in the vector and enabling the rapid removal of the vector after said expression. It was discovered that by adding a micro-RNA target sequence to the NP, P, or L gene of a negative-strand RNA viral vector, it is possible to control the expression of the vector depending on the micro-RNA expressed by the introduction cell. In particular, when a micro-RNA target sequence was added to the NP or P gene, the expression of the vector decreased depending on the micro-RNA, and the removal of the vector was promoted, while the effect was reversed when a micro-RNA target sequence was added to the L gene. The vector can be applied in cell therapy and regenerative medicine and can be used as a therapeutic vector that targets cancer.

Abstract: Disclosed herein are therapeutic applications of CRISPR/Cpf1-based genome editing.

Abstract: Compositions and methods for producing modified AAV Cap genes and combinatorial libraries of chimeric AAV vectors and virions in an AAV serotype 3 background. Selecting for modified AAV3 virions displaying cell- or tissue-specific tropisms differing from WT AAV3. Using the synthetic combinatorial AAV3 capsid libraries for introducing into a selected target host cells one or more nucleic acid molecules useful in diagnostic and/or therapeutic gene-therapy regimens.

Abstract: The present invention relates to a vector for use in the treatment of a polyglutamine repeat spinocerebellar ataxia, which vector comprises cholesterol 24-hydroxylase encoding nucleic acid.

Abstract: Produced the gene therapy DNA vectors based on the gene therapy DNA vector VTvaf17 for the treatment of diseases featuring disruption of mucociliary transport, mucolytic function and development of mucostasis. The gene therapy DNA vector contains the coding region of the SKI, TGFB3, TIMP2 or FMOD therapeutic genes. Methods of producing or use a gene therapy DNA vector based on gene therapy DNA vector VTvaf17 carrying SKI, TGFB3, TIMP2 or FMOD therapeutic genes. The methods of producing strain for production of gene therapy DNA vector for treatment of diseases featuring disruption of mucociliary transport and development of mucostasis. Escherichia coli strain SCS 110-AF/VTvaf17-SKI, SCS 110-AF/VTvaf17-TGFB3, SCS110-AF/VTvaf17-TIMP2 or SCSI 10-AF/VTvaf17-FMOD obtains by the method described above carrying gene therapy DNA vector VTvaf17-SKI, VTvaf17-TGFB3, VTvaf17-TIMP2 or VTvaf17-FMOD.

Abstract: Compositions for the in vivo delivery of a gene editing CRISPR/Cas9 complex was developed to eliminate integrated retroviral DNA sequences from latently infected human cells and animal disease models.

Abstract: The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.

Abstract: The present invention provides a carboxylated nanodiamond-mediated CRISPR-Cas9 delivery system for gene editing comprising nanodiamond (ND) particles as the carriers of CRISPR-Cas9 components designed to introduce the mutation in a given gene for repairing a tissue damage.