Abstract: A method for freeze-drying a hydrogel composition is disclosed, the method comprising providing the hydrogel composition, wherein the hydrogel composition comprises cellulose nanofibrils and/or cellulose nanocrystals, at least one saccharide, at least one amino acid, and biologics; and freeze-drying the hydrogel composition, thereby obtaining a freeze-dried hydrogel composition.

Abstract: The invention provides an animal model for hepatocellular carcinoma and uses thereof. The genome of the animal model includes a hepatitis B virus genome and a single set of endogenous miR-122 that is partially deleted. Due to the early and high incidence of spontaneous hepatocellular carcinoma in the animal model and its abnormal fat metabolism, the animal model can be used to screen candidate agents for prevention or treatment of hepatitis B virus and hepatocellular carcinoma or other diseases or to develop uses for diagnosis and prediction of hepatocellular carcinoma.

Abstract: Systems comprising a microfluidic device are provided for maintaining and analyzing tissue slices. Methods for maintaining tissue slices in a microfluidic device are further provided.

Abstract: The present invention relate to autologous isolated unpolarized human macrophages for use in the treatment of liver disease and macrophages for use in a method of treating fibrosis in a human in need thereof.

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: Kits and methods of using the kits for analyzing macromolecules, including peptides, polypeptides, and proteins, employing nucleic acid encoding are disclosed. The sample analysis kits employ nucleic acid encoding and/or nucleic acid recording of a molecular interaction and/or reaction, such as recognition events (e.g., between an antigen and an antibody, between a modified terminal amino acid residue, or between a small molecule or peptide therapeutic and a target, etc.). Additional barcoding reagents, such as those for cycle-specific barcoding (e.g., “clocking”), compartment barcoding, combinatorial barcoding, spatial barcoding, or any combination thereof, may be included in the kits. The sample may comprise macromolecules, including peptides, polypeptides, and proteins, and the recording may generate molecular interaction and/or reaction information, and/or polypeptide sequence information.

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: The present disclosure relates to a method of assembling long nucleic acids by enzymatically ligating oligonucleotide molecules hybridized to an indexed splint oligonucleotide molecules. Also disclosed are oligonucleotide structures comprising an indexed splint oligonucleotide useful in performing the disclosed method.

Abstract: Methods of gene therapy are provided for treating or preventing age-related diseases or conditions by regulating one or more functional proteins associated with age-related diseases or conditions.

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: In some aspects, the disclosure relates to compositions and methods that are useful for treating inflammatory disorders and conditions. In some embodiments, the disclosure provides recombinant adeno-associated virus (rAAV) vectors and particles encoding a transgene that expresses a Myxomavirus serine proteinase inhibitor (Serp) protein (e.g., Serp-1) and methods for treating inflammatory disorders using the same.

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

Abstract: The present disclosure relates to methods of treating a patient with a cancer by administering to the patient a composition comprising CAR T cells wherein the CAR T cells comprise a CAR and the CAR comprises an E2 anti-fluorescein antibody fragment, and administering to the patient a small molecule linked to a targeting moiety by a linker. The disclosure also relates to compositions for use in such methods.

Abstract: The invention provides improved methods for enriching targeted genomic regions of interest to be analyzed by multiplexed parallel sequencing. The methods of the invention utilize a pool of TArget Capture Sequences (TACS), wherein the pool comprises a plurality of TACS families, each member of a family binding to the same target sequence but with different start and/or stop positions on the sequence (i.e., staggered binding of the family members to the target sequence) to thereby enrich for target sequences of interest, followed by massive parallel sequencing and statistical analysis of the enriched population. The methods of the invention can be used for a variety of clinical purposes, including non-invasive prenatal testing for chromosomal abnormalities, for example using a maternal blood sample or a sample of fetal cells, assessment of maternal and paternal carrier status for genetic disorders and detection of tumor biomarkers (e.g., liquid biopsy).

Abstract: Embodiments of the invention include systems and media for mammalian cell culture that enhance cell viability which enables biological assays and quantitative studies in tissues such as skin explants. Certain embodiments may be used in coordination with each other or may be practiced separately. An exemplary embodiment of the invention is a culture medium that includes Dulbecco's Modified Eagle's Medium, X-VIVO medium, and subcutaneous adipocyte medium. In one embodiment the culture medium includes approximately 30-60% Dulbecco's Modified Eagle's Medium by volume, approximately 30-60% X-VIVO medium by volume, and approximately 2.5-15% subcutaneous adipocyte medium by volume. Certain embodiments of the culture medium can also include recombinant human stem cell factor. Embodiments of the invention disclosed here are not limited to the brands described and encompass equivalents, as well as combinations, of the individual chemical components that make up the media described herein.

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: Disclosed herein are formulations, substrates, and arrays. In certain embodiments, substrates and arrays comprise a porous layer for synthesis and attachment of polymers or biomolecules. Also disclosed herein are methods for manufacturing and using the formulations, substrates, and arrays, including porous arrays. Also disclosed herein are formulations and methods for one-step coupling, e.g., for synthesis of peptides in an N?C orientation. In some embodiments, disclosed herein are formulations and methods for high efficiency coupling of biomolecules to a substrate.

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.