The present invention relates generally to polypeptides or nucleic acids for use in the treatment, management, retardation of progression or normalisation of development of an iduronate-2-sulfatase (IDS) deficiency and/or Mucopolysaccharidosis type II (MPS II) in an individual, wherein the polypeptides comprise iduronate-2-sulfatase (IDS) tethered to a tandem repeat of Apolipoprotein E (ApoEII) or the nucleic acids comprise an iduronate-2-sulfatase (IDS) gene sequence tethered to a tandem repeat of the Apolipoprotein E (ApoEII) gene sequence. The invention also relates to haematopoietic stem and progenitor cells (HSPCs) transduced by such nucleic acids for use in therapies.

The present invention relates generally to polypeptides or nucleic acids for use in the treatment, management, retardation of progression or normalisation of development of an iduronate-2-sulfatase (IDS) deficiency and/or Mucopolysaccharidosis type II (MPS II) in an individual, wherein the polypeptides comprise iduronate-2-sulfatase (IDS) tethered to a tandem repeat of Apolipoprotein E (ApoEII) or the nucleic acids comprise an iduronate-2-sulfatase (IDS) gene sequence tethered to a tandem repeat of the Apolipoprotein E (ApoEII) gene sequence. The invention also relates to haematopoietic stem and progenitor cells (HSPCs) transduced by such nucleic acids for use in therapies.

The present invention provides compositions, systems, kits, and methods for combining a. single myeloma cell and a single B-cell (e.g., from an animal exposed to a desired antigen) via discrete entity (e.g., droplet) microfluidics. In certain embodiments, a microfluidic device is used to merge a discrete entity containing a B-cell, and a discrete entity containing a myeloma cell, and a discrete entity containing gellable material, at a merger region via a trapping element in order to generate a combined discrete entity. In further embodiments, the combined discrete entity is treated such that a gelled discrete entity is formed.

The present disclosure relates to methods for improving myogenic differentiation capacity of a cell line or an immortalized cell line. For example, the present disclosure relates to methods of exposing an immortalized cell line (e.g., an immortalized fibroblast cell line) to culture media comprising signaling pathway agonists, antagonist, or a combination thereof in order to improve differentiation capacity. In another example, the present disclosure relates to methods of improving differentiation capacity of a cell line or an immortalized cell line where the method includes transforming an immortalized cell line with one or more myogenic regulatory factors and exposing the immortalized cell line to culture media comprising signaling pathway agonists, antagonists, or a combination thereof.

The present disclosure relates to methods for improving myogenic differentiation capacity of a cell line or an immortalized cell line. For example, the present disclosure relates to methods of exposing an immortalized cell line (e.g., an immortalized fibroblast cell line) to culture media comprising signaling pathway agonists, antagonist, or a combination thereof in order to improve differentiation capacity. In another example, the present disclosure relates to methods of improving differentiation capacity of a cell line or an immortalized cell line where the method includes transforming an immortalized cell line with one or more myogenic regulatory factors and exposing the immortalized cell line to culture media comprising signaling pathway agonists, antagonists, or a combination thereof.

The present disclosure relates to a method of obtaining a cell where fucosylation pathways are modified, leading to production of partially fucosylated and non-fucosylated protein products, specifically antibodies from the cell. The present disclosure employs the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. The method of the present disclosure targets the Fut8 gene and GMD gene in a cell. Such products are used in developing therapeutics and biomarkers, and in diagnosis and prognosis of diseases.

The present invention relates to a process for producing a composition comprising an aqueous medium and, disposed in the aqueous medium, a first volume of a first hydrogel, which process comprises: (i) providing a composition comprising a first hydrophobic medium and, disposed in the first hydrophobic medium, a first volume of a first hydrogel; (ii) disposing a volume of an aqueous composition comprising a hydrogel compound around the first volume of the first hydrogel; (iii) allowing the aqueous composition comprising the hydrogel compound to form a gel and thereby forming a hydrogel object, which hydrogel object comprises the first volume of the first hydrogel and a second volume of a second hydrogel, which second volume of the second hydrogel is disposed around the first volume of the first hydrogel; and (iv) transferring the hydrogel object from the first hydrophobic medium to an aqueous medium and thereby producing the composition comprising the aqueous medium and, disposed in the aqueous medium, the first volume of the first hydrogel. The invention further provides a hydrogel object, which hydrogel object comprises a first volume of a first hydrogel and a second volume of a second hydrogel, which second volume of the second hydrogel is disposed around the first volume of the first hydrogel.

The present invention relates to a process for producing a composition comprising an aqueous medium and, disposed in the aqueous medium, a first volume of a first hydrogel, which process comprises: (i) providing a composition comprising a first hydrophobic medium and, disposed in the first hydrophobic medium, a first volume of a first hydrogel; (ii) disposing a volume of an aqueous composition comprising a hydrogel compound around the first volume of the first hydrogel; (iii) allowing the aqueous composition comprising the hydrogel compound to form a gel and thereby forming a hydrogel object, which hydrogel object comprises the first volume of the first hydrogel and a second volume of a second hydrogel, which second volume of the second hydrogel is disposed around the first volume of the first hydrogel; and (iv) transferring the hydrogel object from the first hydrophobic medium to an aqueous medium and thereby producing the composition comprising the aqueous medium and, disposed in the aqueous medium, the first volume of the first hydrogel. The invention further provides a hydrogel object, which hydrogel object comprises a first volume of a first hydrogel and a second volume of a second hydrogel, which second volume of the second hydrogel is disposed around the first volume of the first hydrogel.

In one embodiment, the present application discloses a mammalian autologous vaccine or allogeneic vaccine comprising an effective amount of a mammalian induced pluripotent stem cells (iPSCs) obtained by reprogramming of somatic cells from a patient; wherein the autologous vaccine or the allogeneic vaccine expresses a gene selected from the group consisting of ASTE1, BIRC5, CDCA1, CDKN2A, DEPDC1, EGFR, ERBB2, FOXM1, GPC3, HJURP, HSPA8, HSP90B1, IDH1, IDO1, IGF2BP3, IMPS, KIF20A, KIF20B, MELK, MGAT5, NUF2, PMEL, RAS, TAF1B, TOMM34, TTK, TP53, VEGFR1 and VEGFR2; and wherein the autologous vaccine or the allogeneic vaccine induces an immune response from the patient for the treatment of cancer.

In order to improve the efficiency of gene introduction in CAR therapy employing a transposon method, provided is a method for preparing genetically-modified T cells expressing chimeric antigen receptor, comprising: (1) a step of preparing non-proliferative cells which are obtained by stimulating a group of cells comprising T cells using an anti-CD3 antibody and an anti-CD28 antibody followed by a treatment for causing the cells to lose their proliferation capability; (2) a step of obtaining genetically-modified T cells into which a target antigen-specific chimeric antigen receptor gene has been introduced using a transposon method; (3) a step of mixing the non-proliferative cells prepared by step (1) with the genetically-modified T cells obtained by step (2), and co-culturing the mixed cells while stimulating the mixed cells using an anti-CD3 antibody and anti-CD28 antibody; and (4) a step of collecting the cells after culture.