A01K2207/30

METHODS AND MATERIALS FOR REDUCING CARDIAC XENOGRAFT REJECTION

This document provides methods and materials involved in reducing cardiac xenograft rejection. For example, methods and materials for preparing transgenic pigs expressing reduced or no endogenous Sd.sup.a or SDa-like glycans derived from the porcine β1,4 N-acetyl-galactosaminyl transferase 2 (B4GALNT2) glycosyltransferase and/or reduced or no endogenous α-Gal antigens, methods and materials for modifying the xenograft recipient's immunological response to non-Gal antigens (e.g. CD46, CD59, CD9, PROCR, and ANXA2) to reduce cardiac xenograft rejection, and methods and materials for monitoring the progress of xenotransplant immunologic rejection are provided.

COMPOSITIONS AND METHODS FOR TREATING REFRACTORY SEIZURES
20210228518 · 2021-07-29 ·

The present invention relates to the field of seizures. More specifically, the present invention provides compositions and methods for treating refractory seizures in neonates. In one embodiment, the method comprises the steps of (a) administering to the patient an amount of a KCC2 agonist and/or trkB antagonist effective to restore KCC2 expression to normal physiological levels; and (b) administering to the patient an effective amount of an anti-seizure medication.

ANC80 ENCODING SPHINGOLIPID-METABOLIZING PROTEINS
20210244827 · 2021-08-12 ·

The present disclosure pertains to the use of an Anc80 viral vector that encodes a sphingolipid-metabolizing protein such as acid ceramidase to achieve expression of the sphingolipid-metabolizing protein in a mammalian cell or group of cells. Expression of the protein from the Anc80 vector reduces high levels of ceramide in the cell that lead to cell death or senescence.

Gene Editing to Improve Joint Function

The present invention provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.

ESOPHAGEAL TISSUE AND/OR ORGANOID COMPOSITIONS AND METHODS OF MAKING SAME
20210189349 · 2021-06-24 ·

The instant disclosure relates to methods for converting mammalian definitive endoderm (DE) cells into specific tissue(s) or organ(s) through directed differentiation. In particular, the disclosure relates to formation of esophageal tissue and/or organoids formed from differentiated definitive endoderm.

ANABOLIC TARGETING STEM CELL GENE THERAPY FOR OSTEOPOROSIS

In one aspect, anabolic agent fusion proteins and compositions comprising anabolic agent fusion proteins are provided. In some embodiments, the anabolic agent fusion protein comprises a platelet derived growth factor (PDGF) or a fibroblast growth factor (FGF) and an Asp-Ser-Ser tripeptide (DSS) repeat sequence. In another aspect, methods of promoting bone growth and methods of treating a fracture using anabolic agent fusion proteins and compositions comprising anabolic agent fusion proteins are provided.

TREATING SPINAL CORD INJURY (SCI) AND BRAIN INJURY USING GSX1

Methods for treating a neurological disorder, such as a traumatic spinal cord injury or traumatic brain injury, or a disorder such as Parkinson's disease or multiple sclerosis are provided. Such methods include administering a therapeutically effective amount of Gsx1 protein (such as a Gsx1-cell penetrating peptide fusion protein), or a nucleic acid molecule encoding such a protein (for example as part of a viral vector), thereby treating the neurological disorder.

Gene Editing to Improve Joint Function

The present invention provides compositions and methods for treating joint disorders that are characterized by an inflammatory component. In some aspects, the compositions and methods are to prevent the progression of osteoarthritis and other arthritides and to treat osteoarthritis and other arthritides in a mammalian joint.

PREVENTION AND TREATMENT OF OCULAR INFECTION, COMPOSITION AND METHODS THEREOF

The present disclosure relates to a method to reduce virulence of Tsukamurella comprising decreasing the expression of the mycolyltransferase C (‘tmytC”) gene. Also disclosed is a pharmaceutical composition and method of prevention and treatment of infection by inhibition of tmytC.

Therapeutic agent for ischemic diseases

Provided are: a transformation plasmid for transforming anaerobes and enabling highly efficient and stable secretory expression of a target protein; a gene delivery carrier formed from said anaerobes transformed by said plasmid; a pharmaceutical composition including said gene delivery carrier; and a method for diagnosing or treating an ischemic disease utilizing these. Also provided are: a novel secretory signal; a transformation plasmid including said secretory signal; a gene delivery carrier formed from anaerobes transformed by said plasmid; a pharmaceutical composition including said gene delivery carrier; and a method for diagnosing or treating an ischemic disease utilizing these.