C12N5/078

Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction

Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.

ANTI-TCR ANTIBODY MOLECULES AND USES THEREOF

The disclosure provides antibody molecules that bind to TCR Vβ regions and multispecific molecules comprising said antibody molecules. Additionally, disclosed are nucleic acids encoding the same, methods of producing the aforesaid molecules, pharmaceutical compositions comprising aforesaid molecules, and methods of treating a cancer using the aforesaid molecules.

HIV pre-immunization and immunotherapy

The present invention relates generally to immunization and immunotherapy for the treatment or prevention of HIV. In particular, the methods include in vivo and/or ex vivo enrichment of HIV-specific CD4+ T cells.

Monolayer of PBMCs or bone-marrow cells and uses thereof

The invention relates to peripheral blood mononuclear cell (PBMC) monolayers or bone-marrow cell monolayers and methods for its culture and corresponding uses of said monolayers. The present invention also relates, in some aspects, to screening methods comprising the PBMC monolayer or bone-marrow cell monolayer of the invention for determination of response or lack of response of a disease to a therapeutic agent and/or drug screening methods. In some aspects, the invention further relates to methods for diagnosing a disease or predisposition to a disease in a PBMC donor or bone-marrow cell donor comprising the PBMCs/bone-marrow cells cultured according to the method of the invention and/or to methods for determining whether the disease is likely to respond or is responsive to treatment with a therapeutic agent.

MONOLAYER OF PBMCS OR BONE-MARROW CELLS AND USES THEREOF

The invention relates to peripheral blood mononuclear cell (PBMC) monolayers or bone-marrow cell monolayers and methods for its culture and corresponding uses of said monolayers. The present invention also relates, in some aspects, to screening methods comprising the PBMC monolayer or bone-marrow cell monolayer of the invention for determination of response or lack of response of a disease to a therapeutic agent and/or drug screening methods. In some aspects, the invention further relates to methods for diagnosing a disease or predisposition to a disease in a PBMC donor or bone-marrow cell donor comprising the PBMCs/bone-marrow cells cultured according to the method of the invention and/or to methods for determining whether the disease is likely to respond or is responsive to treatment with a therapeutic agent.

BLOOD SUBSTITUTES COMPRISING HEMOGLOBIN AND METHOGS OF MAKING
20230090172 · 2023-03-23 ·

Methods for making hemoglobin based blood substitute preparations and hemoglobin based blood substitute preparations. The methods involve preparing a low purity erythrocyte protein fraction comprising hemoglobin protein and endogenous non-hemoglobin protein complement, and chemically modifying the proteins in the protein fraction to form a cross-linked hemoglobin containing blood substitute preparation. The low purity erythrocyte protein preparation can contain from at least about 0.2% (mole/mole) up to about 20% (mole/mole) endogenous non-hemoglobin protein complement. At least about 90% (mole/mole) of the hemoglobin proteins can be cross-linked, so that the average molecular mass of cross-linked proteins comprising hemoglobin protein molecules in the preparation is at least about 300 kDa. The preparations can be used to prepare finished blood substitute formulations for in-vivo and ex-vivo use.

Use of LRP2 Agonists for Generating Myeloid-Derived Suppressor Cells
20220348869 · 2022-11-03 ·

Provided herein are methods of generating MDSCs ex vivo. The methods include culturing blood cells with an LRP2 agonist.

FETAL CELL CAPTURE MODULE AND MICROFLUIDIC CHIP FOR FETAL CELL CAPTURE AND METHODS FOR USING THE SAME
20230092810 · 2023-03-23 ·

The present invention relates to a fetal cell capture module, a microfluidic chip for fetal cell capture, and methods for using the same. The fetal cell capture module comprises a cell capture carrier and recognition molecule(s) for specific capture the cell(s). The recognition molecule is attached to the surface of the carrier via an organic conjugate L comprising disulfide bonds. The surface of the chip is modified with recognition molecules that specifically capture fetal cells via organic conjugates comprising disulfide bonds. The recognition molecule, after capturing the cell, achieves the release of the cell by chemically cleaving the disulfide bonds in the organic coupling conjugate. The present invention enables the capture of fetal cell(s) from whole blood without pre-treatment with a high capture rate, low cell loss, simple and accurate cell release operation, and the efficient and noninvasive release of fetal cells and whole genome analysis.

METHODS OF ENGINEERING PLATELETS FOR TARGETING CIRCULATING TUMOR CELLS
20220348938 · 2022-11-03 ·

Disclosed herein are nucleic acid constructs that can be used to build genetic circuits for producing antibodies comprising split toxins. Also disclosed herein are methods of producing platelets comprising the antibodies. The platelets produced by the methods disclosed herein can be used to target circulating tumor cells.

PLATELET STORAGE METHODS AND COMPOSITIONS
20230086683 · 2023-03-23 ·

Disclosed are compounds and compositions for slowing, preventing, or reversing platelet damage, particularly as may occur during blood banking or during refrigeration of platelets. Also disclosed herein are methods for storing platelets and methods for improving platelet survival upon transfusion with one or more compounds or compositions as described herein.