C12N2320/31

METHODS FOR AMELIORATING INFUSION REACTIONS

Certain embodiments of the invention provide methods of ameliorating an infusion reaction in a mammal in need thereof.

ENHANCING EXPRESSION OF LINE-1 ENCODED ORF2P FOR CANCER THERAPEUTICS
20230053473 · 2023-02-23 ·

Methods for treating a neoplasia are provided. Further provided are novel targets for cancer chemotherapy including ORF2p Protein and methods for increasing expression of ORF2p in neoplasias. Monoclonal antibodies are also provided to various epitopes of ORF2p and their use in research and diagnostivds.

COMPOSITIONS AND METHODS FOR REPROGRAMMING AGE-RESTRICTED NON-NEURONAL CELLS
20220364091 · 2022-11-17 ·

Provided herein are compositions and methods for reprogramming a non-neuronal cell to a neuron. Aspects of the present disclosure relate to compositions and methods for transdifferentiating an age-restricted non-neuronal cell into a neuron. Also provided herein is a method of treating neurodegenerative disease by reprogramming region or anatomy specific non-neuronal cells into specific types of functionalized neurons.

RNA interference in ocular indications

The present invention relates to ocular administration of sd-rxRNA and rxRNAori molecules.

Methods of treating a retroviral infection

The invention relates to a non-coding sequence of deoxyribonucleic acids comprising at least one sequence motif N.sup.1N.sup.2CGN.sup.3N.sup.4, wherein N is a nucleotide comprising A, C, T, or G, and C is deoxycytidine, G is deoxyguanosine, A is deoxyadenosine and T is deoxy-thymidine for the treatment of viral infections. In particular, the non-coding sequence of deoxyribonucleic acids is used in combination with antiretroviral therapy and/or histone de-acetylase inhibitors.

TUNABLE ANCHOR FOR LIPOSOMAL SPHERICAL NUCLEIC ACID ASSEMBLY
20220364095 · 2022-11-17 ·

The disclosure is generally related to spherical nucleic acids (SNAs), structures comprising a nanoparticle core surrounded by a shell of oligonucleotides. In some aspects the disclosure provides a spherical nucleic acid (SNA) comprising: (a) a nanoparticle core; and (b) a shell of oligonucleotides attached to the nanoparticle core, wherein one or more oligonucleotides in the shell of oligonucleotides is attached to the nanoparticle core through a hydrophobic anchor comprising one or more dodecane (C12) subunits. Methods of making and using the SNAs are also provided herein.

Human antibodies to the human glucagon receptor and methods of use thereof to lower blood glucose or ketone levels

The present invention provides antibodies that bind to the human glucagon receptor, designated GCGR and methods of using same. According to certain embodiments of the invention, the antibodies are fully human antibodies that bind to human GCGR. The antibodies of the invention are useful for lowering blood glucose levels and blood ketone levels and are also useful for the treatment of diseases and disorders associated with one or more GCGR biological activities, including the treatment of diabetes, diabetic ketoacidosis and long-term complications associated with diabetes, or other metabolic disorders characterized in part by elevated blood glucose levels.

Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies

The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).

COMPOSITIONS AND METHODS OF TREATMENT FOR LYTIC AND LYSOGENIC VIRUSES
20230048681 · 2023-02-16 ·

A composition for treating a lysogenic virus, including isolated nucleic acid encoding two or more gene editors chosen from gene editors that target viral DNA, gene editors that target viral RNA, and combinations thereof. A composition for treating a lytic virus, including isolated nucleic acid encoding at least one gene editor that targets viral DNA and a viral RNA targeting composition. A composition for treating both lysogenic and lytic viruses, including isolated nucleic acid encoding two or more gene editors that target viral RNA, chosen from CRISPR-associated nucleases, Argonaute endonuclease gDNAs, C2c2, RNase P RNA, and combinations thereof. A composition for treating lytic viruses, including isolated nucleic acid encoding two or more gene editors that target viral RNA and a viral RNA targeting composition. Methods of treating a lysogenic virus or a lytic virus, by administering the above compositions to an individual having a virus and inactivating the virus.

Immunomodulatory small hairpin RNA molecules

The present invention generally relates to specific immune-modulatory RNA species that have a small hairpin structure (shRNA), and that can bind to retinoic acid inducible gene I receptor (RIG-I). In particular, said RNA species comprise a nucleotide insertion to create a kink in the stem region. Also encompassed are compositions comprising such shRNA, for use as antiviral or anticancer medication, or as adjuvants in vaccine.