C12N2800/206

TRIPLE-MODE SYSTEM FOR ANTIBODY MATURATION, SURFACE DISPLAY AND SECRETION

The present invention provides a triple-mode antibody display system that simultaneously matures, displays and secretes an antibody to a target of interest. An antibody in vivo-matured and complexed with membrane anchored bait can be expressed on the surface of the host cell, while complexed with a soluble bait the antibody is secreted from the host cell. Methods of using the system for identifying binders that bind specifically to an antigen of interest are also provided. Polypeptides, polynucleotides and host cells useful for making the protein binder display system are also provided along with methods of use thereof

COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION
20240301411 · 2024-09-12 · ·

Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APP RNA in a cell or a subject, and in certain instances reducing the amount of APP protein in a cell or a subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease or disorder associated with APP. Such symptoms and hallmarks include cognitive impairment, including a decline in memory and language skills, behavioral and psychological symptoms such as apathy and lack of motivation, gait disturbances, seizures, progressive dementia, and abnormal amyloid deposits.

MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES

The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

Modulation of BCL11A for treatment of hemoglobinopathies

The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

Ungulates with Genetically Modified Immune Systems
20170183685 · 2017-06-29 ·

The present invention provides ungulate animals, tissue and organs as well as cells and cell lines derived from such animals, tissue and organs, which lack expression of functional endogenous immunoglobulin loci. The present invention also provides ungulate animals, tissue and organs as well as cells and cell lines derived from such animals, tissue and organs, which express xenogenous, such as human, immunoglobulin loci. The present invention further provides ungulate, such as porcine genomic DNA sequence of porcine heavy and light chain immunoglobulins. Such animals, tissues, organs and cells can be used in research and medical therapy. In addition, methods are provided to prepare such animals, organs, tissues, and cells.

MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES

The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

Ungulates with genetically modified immune systems
09585374 · 2017-03-07 · ·

The present invention provides ungulate animals, tissue and organs as well as cells and cell lines derived from such animals, tissue and organs, which lack expression of functional endogenous immunoglobulin loci. The present invention also provides ungulate animals, tissue and organs as well as cells and cell lines derived from such animals, tissue and organs, which express xenogenous, such as human, immunoglobulin loci. The present invention further provides ungulate, such as porcine genomic DNA sequence of porcine heavy and light chain immunogobulins. Such animals, tissues, organs and cells can be used in research and medical therapy. In addition, methods are provided to prepare such animals, organs, tissues, and cells.