C12N5/0686

Compositions and methods of treatment

The present disclosure provides infectious recombinant adeno-associated virus (rAAV) virions that comprise a variant capsid protein and a heterologous nucleic acid. The present disclosure further provides the variant adeno-associated virus (AAV) capsid proteins (and/or a nucleic acid encoding the variant AAV capsid proteins), which confer to an infectious rAAV virion an increased resistance to human AAV neutralizing antibodies. The present disclosure further provides host cells comprising an infectious rAAV virion and/or a nucleic acid encoding a subject variant AAV capsid protein. The present disclosure further provides methods of delivering a heterologous nucleic acid to a target cell where the target cell is contacted with a subject infectious rAAV virion. The present disclosure further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.

MULTISPECIFIC TRANSTHYRETIN IMMUNOGLOBULIN FUSIONS
20220324924 · 2022-10-13 · ·

The present invention relates to multispecific transthyretin (TTR) complexes useful as multispecific binding proteins. The multispecific TTR complexes described herein are particularly useful in binding to one, two, or more epitopes which may be present on one or more proteins. Methods for treating diseases using the TTR complexes of the present invention are described herein.

Method for acclimating and suspending Vero and second order production process for virus
11629338 · 2023-04-18 · ·

Disclosed is a second-order culture method for producing a suspended Vero cell vaccine, wherein same comprises the following steps: suspension culture growth of Vero cells, inoculation of a virus, dilution or addition of a production medium, secondary growth of cells, and optionally harvesting and purifying the virus fluid to produce the vaccine.

METHOD FOR ULTRA-RAPIDLY SELECTING SIGNAL PEPTIDE TO WHICH INDIVIDUAL BARCODE SYSTEM FOR INCREASING PROTEIN PRODUCTIVITY IS INTRODUCED

The present invention relates to a composition for screening various signal peptides to select specific ones that allow efficient secretion of a target protein to out of host cells. The present invention also relates to a method for selecting specific signal peptides that express a target protein in host cells and efficiently secrete the target protein to out of the host cells. The use of the composition and/or method according to the present invention enables ultrafast selection of optimal signal peptides for a target protein through barcoding sequences corresponding to the signal peptides, leading to the maximization of the production yield of the recombinant protein.

MDBK IRF3/IRF7 KNOCK OUT MUTANT CELL AND ITS USE FOR VACCINE PRODUCTION

The present invention pertains to a Madin-Darby bovine kidney (MDBK) cell, wherein the interferon regulatory factors (IRF) IRF3 and/or IRF7 encoding genes are functionally inactivated. The invention also pertains to a cell culture comprising the MDBK cell, use of the MDBK cell culture, a method for the production of a virus using the cell and a vaccine prepared by using the cell.

ULTRASONIC GENETICALLY ENCODED CALCIUM INDICATORS

Disclosed herein include methods, compositions, and kits suitable for use in calcium imaging. There are provided, in some embodiments, Ca.sup.2+-sensing GvpC proteins. Disclosed herein include Ca.sup.2+-sensing gas vesicles (GVs) comprising Ca.sup.2+-sensing GvpC proteins. In some embodiments, the Ca.sup.2+-sensing GvpC protein is capable of undergoing a first allosteric conformational change upon the Ca.sup.2+-binding domain binding Ca.sup.2+ that causes the Ca.sup.2+-sensing GV to change from a GV stiff state to a GV soft state. One or more of the mechanical, acoustic, surface, and magnetic properties of a Ca.sup.2+-sensing GV can differ between the GV soft state and the GV stiff state.

KIDNEY ORGANOIDS AND METHOD FOR PRODUCING THE SAME
20230142476 · 2023-05-11 ·

Disclosed is a method for producing kidney organoids including steps of: (1) differentiating stem cells into metanephric mesenchyme cells; (2) forming metanephric mesenchyme cell aggregates by culturing the metanephric mesenchyme cells; and (3) differentiating the metanephric mesenchyme cell aggregates into kidney organoids.

TRIPLY PERIODIC MINIMAL SURFACES FOR 3D PRINTED ORGANS AND TISSUES
20230143576 · 2023-05-11 · ·

An apparatus can include a triply periodic minimal surface. The apparatus can include a 3D scaffold formed from the triply periodic minimal surface. The apparatus can include one or more channels formed by the 3D scaffold. A method of forming a gas exchange unit can include printing a 3D scaffold formed from a triply periodic minimal surface. The 3D scaffold can include a vascular network configured to conduct a fluid. The 3D scaffold can include one or more channels configured to hold a gas. The vascular network can be embedded inside walls of the 3D scaffold. The one or more channels can be positioned between the walls of the 3D scaffold.

LONG-TERM CELL-CULTIVATION USING POLYIMIDE POROUS MEMBRANE AND CELL-CRYOPRESERVATION METHOD USING POLYIMIDE POROUS MEMBRANE
20170369838 · 2017-12-28 ·

The invention relates to a long term cell culturing method and a cell culturing apparatus and kit that employ a porous polyimide film. The invention further relates to a cell cryopreservation method and kit employing the porous polyimide film.

GENE EDITING SYSTEMS COMPRISING AN RNA GUIDE TARGETING STATHMIN 2 (STMN2) AND USES THEREOF

A system for genetic editing of a stathmin 2 (STMN2) gene, comprising (i) a Cas12i2 polypeptide or a first nucleic acid encoding the Cas12i2 polypeptide, and (ii) an RNA guide or a second nucleic acid encoding the RNA guide, wherein the RNA guide comprises a spacer sequence specific to a target sequence within an STMN2 gene. Also provided herein are methods for editing a STMN2 gene using the gene editing system disclosed herein and/or for treating diseases associated with the STMN2 gene.