C12N5/0647

CELLS WITH INCREASED IMMUNO-REGULATORY PROPERTIES AND METHODS FOR THEIR USE AND MANUFACTURE

The present invention is directed to compositions and methods to increase the expression of PD-L1 and/or IDO-1 in a population of cells, the modulated cells expressing increased PD-L1 and/or IDO-1, and methods related to the immunosuppressive effects obtained by cells expressing increased PD-L1 and/or IDO-1.

PERIPHERAL BLOOD DERIVED SMALL PLURIPOTENT CELLS
20230235288 · 2023-07-27 ·

The present disclosure relates to populations of small pluripotent stem cells derived from peripheral blood, such as human peripheral blood. In some aspects, these small pluripotent stem cells are smaller than known stem cells and express a range of embryonic, hematopoietic, or mesenchymal stem cell markers. Also disclosed herein are methods of isolation and cryopreservation of these populations of small pluripotent stem cells. These small pluripotent stem cells may be differentiated in a wide range of cell types, which can be used in various applications such as the study of cell activity or for treatment of diseases and personalized medicine.

PERMEATION OF WHOLE VERTEBRAL BODIES WITH A CRYOPROTECTANT USING VACUUM ASSISTED DIFFUSION
20230232820 · 2023-07-27 ·

The present disclosure relates to, at least, a vacuum-assisted method for infiltrating cadaver bone with a cryoprotectant and a method for rapidly warming the cryopreserved cadaver bone for bone marrow processing.

THYMUS ORGANOIDS BIOENGINEERED FROM HUMAN PLURIPOTENT STEM CELLS
20230002727 · 2023-01-05 ·

This document relates to bioengineering and involves bioengineered thymus organoids and related humanized animal models. The thymus organoids and animal models have various commercial and clinical uses, including generating humanized antibodies, making antigen-specific human T cells, inducing transplantation tolerance, rejuvenating thymus functions, and modeling human diseases.

Hemangio-colony forming cells

Methods of generating and expanding human hemangio-colony forming cells in vitro and methods of expanding and using such cells are disclosed. The methods permit the production of large numbers of hemangio-colony forming cells as well as derivative cells, such as hematopoietic and endothelial cells. The cells obtained by the methods disclosed may be used for a variety of research, clinical, and therapeutic applications.

Materials and methods for treatment of hemoglobinopathies

Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for creating permanent changes to the genome that can result in at least one deletion, insertion, modulation, or inactivation of a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

PHARMACEUTICAL COMPOSITION FOR TREATING CHRONIC STROKE

The present invention provides a pharmaceutical composition for treating chronic stroke, involving injection via brain into the cranium of a patient having chronic stroke for six months or more; the pharmaceutical composition is a suspension at least comprising TS stem cells, an active synergistic component and a growth factor, wherein the expression level of CD34 and CD45 of the TS stem cells is 10% or less, and the expression level of CD90 and CD105 is 90% or more; the active synergistic component is an extracellular vesicle; the growth factor is at least one selected from the group consisting of HGF, G-CSF, Fractalkine, IP-10, EGF, IL-1α, IL-1β, IL-4, IL-5, IL-13, IFNγ, TGFα and sCD40L. The present invention overcomes the limitations of previous cell therapy and provides a cell-based preparation that is clinically safe and therapeutically effective for chronic cerebral stroke.

COMPOSITIONS AND METHODS FOR EDITING BETA-GLOBIN FOR TREATMENT OF HEMAGLOBINOPATHIES

The disclosure features methods of correcting a mutation in the human beta-globin (HBB) gene in a cell or population of cells. The disclosure also features methods of increasing repair of a DNA double stranded break (DSB) in an HBB gene by the homology-directed repair (HDR) pathway. The disclosure also features compositions for use in the methods.

SPLIT DEAMINASE BASE EDITORS
20230024833 · 2023-01-26 ·

Provided herein are compositions and methods for improving the genome-wide specificities of targeted base editing technologies.

SMALL MOLECULE COMPOUNDS FOR AMPLIFYING HEMATOPOIETIC STEM CELLS, AND COMBINATION THEREOF

Provided are small molecule inhibitors for amplifying hematopoietic stem cells (HSCs) and a combination thereof. The small molecule inhibitors and the combination thereof can maintain the sternness of hematopoietic stem cells while promoting the in vitro amplification of hematopoietic stem cells (HSCs).