Abstract: The present invention relates to compositions comprising induced T regulatory cells (iTregs), methods of making the compositions, and methods of using the compositions for enhancing bone remodeling in the treatment of Osteogenesis Imperfecta (OI).

Abstract: This disclosure relates to methods of treatment using compound (1) or analogs thereof, and pharmaceutically acceptable salts thereof. Also disclosed are compounds of formula (10): as defined in the specification, and pharmaceutically acceptable salts thereof, as well as pharmaceutical compositions comprising the same. Methods of treatment, such as for cancer, are provided that comprise administering the compounds and their salts to a subject in need of such treatment.

Abstract: Methods are provided herein for selectively killing senescent cells and for treating senescence-associated diseases and disorders by administering a senolytic agent. Senescence-associated diseases and disorders treatable by the methods using the senolytic agents described herein include cardiovascular diseases and disorders associated with or caused by arteriosclerosis, such as atherosclerosis; idiopathic pulmonary fibrosis; chronic obstructive pulmonary disease; osteoarthritis; senescence-associated ophthalmic diseases and disorders; and senescence-associated dermatological diseases and disorders.

Abstract: Described herein are methods for treating or preventing a disease or disorder of the pulmonary system (e.g., cystic fibrosis), respiratory or digestive system in a subject, the methods comprising administering compounds or compositions comprising water soluble polyglucosamine and derivatized polyglucosamine.

Abstract: A method for producing uniform-size liposomes is provided. The liposomes are coated with a sorting agent to yield a plurality of density-modified liposomes of different sizes. These liposomes are then separated using a densitometric method. The sorting agent includes both a density-modifying moiety and a targeting moiety.

Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of PHD2 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against PHD2 gene expression.

Abstract: Described herein are methods and culture medium, useful for inducing polarization in multipotent stem cells. Additionally, described herein are multipotent cells produced by the methods and culture medium of this disclosure that are useful therapeutic agents. Also described herein are extracellular vesicles and factors secreted by multipotent cells that are produced by the methods and culture medium of this disclosure that are useful as therapeutic agents.

Abstract: The present invention relates to mitochondrial compositions and therapeutic methods of using same. The invention discloses compositions of partially purified functional mitochondria and methods of using the compositions to treat conditions which benefit from increased mitochondrial function by administering the compositions to a subject in need thereof.

Abstract: Methods of minimizing dysregulation of Staufen1-associated RNA metabolism can include introducing an amount of a Staufen1-regulating agent to a target cell sufficient to minimize the dysregulation. Therapeutic compositions for treating a neurodegenerative condition associated with Staufen1-induced dysregulation of RNA metabolism can include a therapeutically effective amount of a Staufen1-regulating agent and a pharmaceutically acceptable carrier.

Abstract: Described herein are block copolymers, and methods of making and utilizing such copolymers. The described block copolymers are disruptive of a cellular membrane, including an extracellular membrane, an intracellular membrane, a vesicle, an organelle, an endosome, a liposome, or a red blood cell. Preferably, in certain instances, the block copolymer disrupts the membrane and enters the intracellular environment. In specific examples, the block copolymer is endosomolytic and capable of delivering an oligonucleotide (e.g., an mRNA) to a cell. Compositions comprising a block copolymer and an oligonucleotide (e.g., an mRNA) are also disclosed.

Abstract: A method of preventing or treating a subject suffering from a flavivirus infection by administering an effective amount of berbamine or its analogue to the subject, berbamine has a structure of Formula (I), wherein the flavivirus infection is caused by Japanese encephalitis virus, Zika virus or Dengue virus. A method of inhibiting the entry of a flavivirus, an enterovirus and/or a lentivirus into host cells includes contacting the host cells with an effective amount of berbamine of its analogue, berbamine has a structure of Formula (I), wherein the flavivirus is Japanese encephalitis virus, Zika virus or Dengue virus.

Abstract: Retrotransposons, operating though human-specific neurological pathways, can contribute to environment, lifestyle, and/or age-related neurodegeneration by disrupting functional mitochondrial populations within neurons. The mitochondrial disruption can occur through a number of retrotransposon-induced mechanisms that can influence the efficient and accurate transcription and/or translation of mitochondrial genes encoded in the nuclear genome, operating primarily through epigenetic processes. Alu element-related conformational changes (both subtle and major) of the outer and inner mitochondrial membrane pores can restrict or prevent the normal translocation of proteins (i.e., TOMM and TIMM complexes), ultimately contributing to mitochondrial stress, mitophagy, inflammation, and neuron and glial cell death.

Abstract: The invention relates to an allele specific siRNA able to silence the expression of only one allele of a heterozygous DNM2 gene, for treating diseases caused by heterozygous mutation and/or overexpression of Dynamin 2.

Abstract: An antibody or fragment thereof, against presenilin, and more specifically against the luminal loop 1 of presenilin, for use in the treatment of cancer, by way of administrating a therapeutically effective quantity of the antibody or a fragment thereof, or a pharmaceutical composition comprising thereof, to a subject who suffers from cancer.

Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a RRM2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a RRM2 gene using said pharmaceutical composition; and methods for inhibiting the expression of RRM2 in a cell.

Abstract: Antisense oligonucleotides target the mutation in intron 26 of the CEP290 gene and reduce inclusion of the aberrant exon into the CEP290 mRNA. The oligonucleotides include no more than 3 consecutive guanosines, have no more than 60% guanosine nucleobases, include at most one CpG sequence, and/or do not have the potential to form a hairpin comprising 3 or more consecutive complementary base pairs.

Abstract: Provided are T cell receptors (TCR) and TCR variable regions that can selectively bind the T-cell leukemia/lymphoma 1 (TCL1) oncoprotein. The TCR may be utilized in various therapies, such as autologous TCL1-TCR adoptive T cell therapy, to treat a cancer, such as a B-cell malignancy or a solid tumor expressing TCL1. Methods for expanding a population of T cells that target TCL1 are also provided.

Abstract: The disclosure provides compositions and methods of treating diseases and conditions associated with phospholipase D (PLD) toxin. In particular, the methods include administering to a subject in need a pharmaceutical composition comprising a dicarboxylic acid ester.

Abstract: The present invention is related to compositions and methods for activating Lck, including in vivo, in vitro and ex vivo uses.

Abstract: The disclosure provides compositions and methods of inducing an analgesic effect. In particular, the methods include administering to a subject in need a pharmaceutical composition comprising a dicarboxylic acid ester.

Abstract: The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.

Abstract: The present invention provides compositions and methods for stimulating an immune response using cationic lipids alone or in combination with antigens.

Abstract: The present disclosure relates to bifunctional chemical epigenetic modifiers, and methods of making, kits and using the bifunctional chemical epigenetic modifiers. The bifunctional chemical epigenetic modifiers can include a FK506 molecule or derivative thereof, a linker and a bifunctional ligand. The bifunctional ligand can be a histone deacetylase inhibitor.

Abstract: The invention pertains to the field of adoptive cell immunotherapy. It provides with engineered immune cells comprising genetic alteration into genes which are involved into immune functions downregulation, especially in response to environment signals such as nutrients depletion. Such method allows the production of more potent immune cells in the context of tumors' microenvironment.

Abstract: A high-resolution dual-beam counter propagating optical-tweezers instrument was designed that can measure forces at <1 pN and one nanometer distance at a temporal resolution of 25 ?s with high accuracy and precision. Using the high-resolution optical-tweezers, time-dependent conformational switching and structural rearrangements in a single-molecule of the guanine aptamer were identified that follow a modified induced-fit model, where guanine remodels multiple barriers and triggers the receptor conformation rapidly to synchronize with the elongating transcriptional machinery for controlling gene regulation.

Abstract: A nanocarrier containing a combination of bioactive agents is provided. The nanocarrier allows for targeted delivery of the agents to cancer cells with minimized side effect.

Abstract: Methods of producing T cells having reduced surface fucosylation and use thereof in adoptive cell therapy, in particular, in cancer treatment are provided.

Abstract: Methods and compositions for treating cancer are disclosed.

Abstract: Fabricating a nanopore sensor includes depositing a first and second oxide layers on first and second sides of a sapphire substrate. The second oxide layer is patterned to form an etch mask having a mask opening in the second oxide layer. A crystalline orientation dependent wet anisotropic etch is performed on the second side of the sapphire substrate using the etch mask to form a cavity having sloped side walls through the sapphire substrate to yield an exposed portion of the first oxide layer, each of the sloped side walls being a crystalline facet aligned with a respective crystalline plane of the sapphire substrate. A silicon nitride layer is deposited on the first oxide layer. The exposed portion of the first oxide layer in the cavity is removed, thereby defining a silicon nitride membrane in the cavity. An opening is formed through the silicon nitride membrane.

Abstract: Systems and methods for predicting a patient response to various agents and/or combinations of agents using ex vivo dosing and imaging are disclosed. In one example, a method of determining treatment efficacy includes analyzing a solid cell culture over time, e.g., first and second responses to a solid cell culture to respective treatments may be compared to determine a treatment efficacy of each treatment. Systems and methods for applying the treatments to the cell culture and analyzing the cell culture and efficacy are disclosed.

Abstract: It is an object to provide a technique useful for embryo transfer and development of reproductive medical care. Provided are a sperm activator containing a disrupted product of one or more cells selected from the group consisting of adipose tissue-derived stem cells, dental pulp-derived stem cells, bone marrow-derived stem cells, and umbilical cord blood-derived stem cells as an active ingredient, and an artificial insemination method utilizing the same.

Abstract: Provided herein are compositions, methods and kits for treating inflammatory bowel disease (IBD) such as Crohn's disease and ulcerative colitis in a mammal in need thereof. The method include administering to a subject with IBD a combination therapy containing a therapeutically effective amount of a chemokine receptor 9 (CCR9) inhibitor compound and a therapeutically effective amount of an anti-IL-23 antibody. Also provided herein is a kit containing the CCR9 inhibitor compound and anti-IL-23 antibody.

Abstract: Methods and compositions for characterizing a target polynucleotide, including, characterizing the sequence of the target polynucleotide, using the fractional translocation steps of the target polynucleotide's translocation through a pore.

Abstract: The invention pertains to the field of adaptive cell immunotherapy. It provides with the genetic insertion of exogenous coding sequence(s) that help the immune cells to direct their immune response against infected or malignant cells. These exogenous coding sequences are more particularly inserted under the transcriptional control of endogenous gene promoters that are sensitive to immune cells activation. Such method allows the production of safer immune primary cells of higher therapeutic potential.

Abstract: Culture media, which contain albumin carrying a reduced amount of fatty acid, are useful for culturing stem cells.

Abstract: A microfluidic cell culture chip which contains a central module comprising a central unit, which contains a support consisting of a non-resorbable membrane, a 3D nanostructured porous membrane, comprising at least one protuberance, and the 3D nanostructured porous membrane and that at least one protuberance being composed of materials suitable for the culture of two distinct cell types; a base, and the central unit being integrated in the base, and forming a whole with the base.

Abstract: The invention provides compositions featuring TRP-4 polypeptides and polynucleotides, methods for expressing such polypeptides and polynucleotides in a cell type of interest, and methods for inducing the activation of the TRP-4 polypeptide in neurons and other cell types using ultrasound.

Abstract: Systems and methods for predicting a patient response to various agents and/or combinations of agents using ex vivo dosing and imaging are disclosed. In one example, a method of determining treatment efficacy includes analyzing a solid cell culture over time, e.g., first and second responses to a solid cell culture to respective treatments may be compared to determine a treatment efficacy of each treatment. Systems and methods for applying the treatments to the cell culture and analyzing the cell culture and efficacy are disclosed.

Abstract: This disclosure features structurally-stabilized and/or cysteine-reactive peptide inhibitors for selective targeting of BFL-1, or dual targeting of BFL-1 and MCL-1. Also disclosed are methods of using such structurally-stabilized and cysteine-reactive peptides in the treatment of BFL-1- and/or MCL-1-expressing or -dependent cancers or diseases of cellular excess (e.g., autoimmune or inflammatory conditions). Also provided are combination therapies comprising such structurally-stabilized and/or cysteine-reactive peptides and inhibitors of the DNA damage response pathway, such as an ATM kinase inhibitor, ATR kinase inhibitor, CHK1/2 inhibitor, or PARP inhibitor; or an inhibitor of MCL-1, or a selective inhibitor of BCL-2, or an inhibitor of BCL-2/BCL-XL, for the treatment of BFL-1-expressing or -dependent cancers (e.g., AML), BFL-1 and MCL-1-expressing or -dependent cancers, or diseases of cellular excess (e.g., autoimmune or inflammatory conditions).

Abstract: Methods of expanding a natural killer (NK) cell fraction for transplantation into a subject are provided, and particularly, methods for providing transplantable NK cell fractions and protocols for their use, which can be employed for applications in cell transplants and infusions for treatment of cancer and other disease.

Abstract: Autologous bone marrow cells (BMC) are transplanted to a heterologous site in a patient after a sample of the patient's BMC has been tested and found to have a phenotypic profile which meets minimum criteria for transplantation. The phenotypic profile may be obtained by screening a sample of bone marrow cells (BMC) from the patient for the phenotypic profile, such as a CD profile, the phenotype profile may be assessed to determine the likelihood that the BMC will be suitable for transplantation to the heterologous tissue site without enriching particular phenotypic population(s) of the BMC.

Abstract: Disclosed are UAP inhibitors to inhibit glucose flux in the hexosamine biosynthetic pathway and methods of treating a disease using the inhibitors.

Abstract: The invention is directed to a more efficient lentiviral vector comprising a nucleic acid sequence encoding a human ?-globin protein or a human ?-globin protein, which is oriented from 5? to 3? relative to the lentiviral genome. The invention also provides a composition and method utilizing the lentiviral vector.

Abstract: Method and apparatus for treating peripheral olfactory dysfunction are described herein. One method may include introducing a treatment device into a nasal cavity of the patient, the treatment device having a proximal end, a distal end, an elongated shaft therebetween, and a treatment end effector disposed on or near the distal end. The distal end of the treatment device may be advanced into proximity of a cribriform plate within the nasal cavity and at least one olfactory neuron may be ablated through the cribriform plate via the treatment end effector to reduce at least one symptom of olfactory dysfunction.

Abstract: The present invention is directed to pemetrexed formulations comprising a non-aqueous solvent that remains stable after dilution for at least about 48 hours when stored at 2° C. to 8° C. The present invention is also directed to pemetrexed formulations comprising a non-aqueous solvent that remains stable for at least about 24 months when stored at 2° C. to 8° C.

Abstract: The present invention is directed to anti-PVRIG antibodies and methods of using same.

Abstract: The present disclosure provides small hairpin nucleic acid molecules capable of stimulating interferon production. The nucleic acid molecules of the present disclosure has a double-stranded section of less than 19 base pairs and at least one blunt end. In certain embodiments, the molecule comprises at least one 5?-triphosphate and/or at least one 5?-diphosphate. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection, and/or ameliorating, minimizing, reversing, and/or preventing persistent SARS-CoV-2 viral infection, and/or minimizing or preventing SARS-CoV-2 viral infection-derived mortality and/or lethality, in a subject. In certain embodiments, compounds and/or compositions of the disclosure are useful for treating, ameliorating, and/or preventing SARS-CoV-2 viral infection in a tumor-bearing subject.

Abstract: The invention provides for systems, methods, and compositions for targeting nucleic acids. In particular, the invention provides non-naturally occurring or engineered RNA-targeting systems comprising a novel RNA-targeting CRISPR effector protein and at least one targeting nucleic acid component like a guide RNA.

Abstract: A method is provided for prognosing a risk in a patient diagnosed with a premalignant lesion of the breast of the lesion progressing to breast cancer, the method comprising: providing a tissue sample of the premalignant lesion; detecting one or more variants of Osteopontin (OPN) selected from OPN-a, OPN-b and OPN-c in the sample; and prognosing an elevated risk of the lesion progressing to breast cancer if OPN-b and/or OPN-c is detected and/or OPN-a is elevated above normal levels in the tissue sample. Methods of assessing risk of death from breast cancer, methods of treatment, and kits for prognosis are also provided herein.

Abstract: The present disclosure relates to compositions, such as siRNA molecules and FN3 domains conjugated to the same, as well as methods of making and using the molecules.