C12N2799/025

ADENO-ASSOCIATED VIRUS CAPSID PROTEINS

The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.

Satiation peptide administration

Disclosed herein are compositions and methods for treating obesity involving satiation gut peptide administration to the mouth of a subject for a predetermined dose and frequency. In other embodiments, materials and methods of treating certain psychological disorders are disclosed involving satiation gut peptides. In exemplary embodiments, the satiation gut peptide pertains to PYY.

Vectors encoding rod-derived cone viability factor
10040835 · 2018-08-07 · ·

The present invention relates to nucleic acids coding for and capable of expressing a rod-derived cone viability factor (RdCVF) and viral vectors containing these nucleic acids. The invention also relates to compositions and pharmaceutical preparations comprising these nucleic acids or vectors, methods of producing or secreting an RdCVF, and methods of treatment.

Method for treating melanoma using a herpes simplex virus and an immune checkpoint inhibitor
10034938 · 2018-07-31 · ·

The invention relates to methods of treating melanoma using a herpes simplex virus in combination with an immune checkpoint inhibitor.

Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies

The invention provides methods to inhibit or treat brain cancers by locally inhibiting expression or activity of growth factors or growth factor receptors.

Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases

The present invention provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of genetic diseases that affect the cone cells of the retina.

Optically-controlled CNS dysfunction

Provided herein are animals expressing light-responsive opsin proteins in the basal lateral amygdala of the brain and methods for producing the same wherein illumination of the light-responsive opsin proteins causes anxiety in the animal. Also provided herein are methods for alleviating and inducing anxiety in an animal as well as methods for screening for a compound that alleviates anxiety in an animal.

Modified viral structural protein with antiviral activity

This disclosure provides a novel strategy to cope with chronic virus infection by introducing a dominant negative viral structural protein to disturb effective virion production. The dominant negative structural protein mimics antiviral drugs through structural and biochemical interactions during virus assembly. An effective gene therapy model for chronic viral infected diseases is proposed in this disclosure, as represented by HBV Cpdominant1 to clear viral infection.

NOVEL THERAPEUTICAL TOOLS AND METHODS FOR TREATING BLINDNESS

The present invention relates to the use of an isolated nucleic acid molecule comprising a nucleotide sequence coding for a hyperpolarizing light-gated ion channel or pump gene from an archeon or for a light-active fragment of said gene, or the nucleotide sequence complementary to said nucleotide sequence, for treating or ameliorating blindness. The light-gated ion channel or pump gene can be a halorhodopsin gene.

SERCA2 THERAPEUTIC COMPOSITIONS AND METHODS OF USE

The present invention provides methods for treating pulmonary hypertension in a subject by delivering a therapeutic adeno-associated virus (AAV)-SERCA2 composition to a subject in need thereof.