University of Florida Research Foundation Inc

University of Pennsylvania

Louisiana State University and Agricultural & Mechanical College

Alfred Samuel Lewin

William W Hauswirth

Cristhian J Ildefonso

Qiuhong Li

Gregory Scott Schultz

Gustavo D Aguirre

David Clair Bloom

William Beltran

Samuel Jacobson

Sonal Sanjeev Tuli

Artur V Cideciyan

Sanford Leon Boye

Alexandra Rose Lucas

Donna M Neumann

Zachary L Watson

Jia Liu

Wen-Tao Deng

Michael T Massengill

Douglas Grant McFadden

Mohammed Masmudur Rahman

Sanford L Boye

Xiaoping Qi

Sonal Sanjeen Tuli

Kimberly Drenser

Timothy D Blalock

Artur Cideciyan

Samuel G Jacobson

Douglas Grant Mcfadden

The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or ocular inflammation. In an exemplary embodiment, a method is disclosed that involves administering an expression vector that delivers a secretable and cell penetrating CARD to a subject in need of treatment or prevention of age-related macular degeneration or another condition involving macular degeneration or ocular neovascularization.

Delivery of card protein as therapy for occular inflammation

In some aspects, the disclosure relates to compositions and methods that are useful for treating inflammatory disorders and conditions. In some embodiments, the disclosure provides recombinant adeno-associated virus (rAAV) vectors and particles encoding a transgene that expresses a Myxomavirus serine proteinase inhibitor (Serp) protein (e.g., Serp-1) and methods for treating inflammatory disorders using the same.

Recombinant adeno-associated viruses encoding serpin peptides and uses thereof

Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering RNA to a subject in order to reduce expression of one or both alleles of an endogenous RHO gene (for example a mutant rho allele associated with retinitis pigmentosa) in a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.

Compositions and methods for treatment of dominant retinitis pigmentosa

Aspects of the application relate to methods and compositions for delivering therapeutic nucleic acids to neural cells or tissue in a subject. Additional aspects of the application relate to therapeutic nucleic acids, for example therapeutic ribozymes, that are useful for inhibiting viral reactivation in a subject.

Method for delivering RNA to neurons to treat herpes infections

The present invention provides methods and compositions for treating and/or preventing age related macular degeneration and other conditions involving macular degeneration, ocular neovascularization, or inflammation, including ocular inflammation. In some embodiments, the methods comprise administering an expression vector that delivers a secretable and cell penetrating Nrf2 to a subject in need thereof.

Delivery of NRF2 as therapy for protection against reactive oxygen species

Delivery of card protein as therapy for ocular inflammation

Disclosed are methods and compositions for preventing, treating, and/or ameliorating one or more symptoms of inflammation in a mammal. In particular, viral vectors and medicaments containing them are disclosed, which are useful in the prophylaxis, therapy, or amelioration of symptoms of one or more inflammatory-mediated mammalian diseases, such as age-related macular degeneration (AMD), arthritis, Bechet's disease, Best macular dystrophy, corneal inflammation, diabetic retinopathy, drusen formation, dry AMD, dry eye, geographic atrophy, glaucomaocular neovascularization, Lupus erythematosus, macular degeneration, Mallatia Leventinese and Doyne honeycomb retinal dystrophy, nephritis, ocular hypertension, ocular inflammation, recurrent uveitis, Sorsby fundus dystrophy, vasculitis, vitreoretinopathy, wet AMD, or related disorders. In exemplary methods, administration of a pharmaceutical composition comprising a recombinant viral vector that delivers a secretable and cell-penetrating M013 protein or peptide to a subject in need thereof facilitated treatment of particular human disorders such as AMD, ocular neovascularization, uveitis, and related inflammatory ocular disease.

Use of AAV-expressed M013 protein as an anti-inflammatory therapeutic

The present invention is directed to a delivery vector for transferring a small peptide coding sequence to a cell for expression of the small peptide coding sequence within the cell. The delivery vector comprises a secretory signal sequence; a sequence encoding a carrier protein operatively associated with the secretory signal sequence; a sequence encoding a cleavage site operatively associated with the sequence encoding a carrier protein; and a sequence encoding a small peptide operatively associated with the sequence encoding a cleavage site.

Small angiotensin peptide expression system in mammalian cells

Aspects of the disclosure relate to methods and compositions for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering nucleic acid (for example an interfering RNA) to a subject in order to reduce expression of one or both alleles of an endogenous rho gene (for example a mutant rho allele associated with retinitis pigmentosa) in the subject. In some embodiments, a replacement rho gene that is resistant to the interfering nucleic acid also is delivered to the subject.

AAV vectors for treatment of dominant retinitis pigmentosa

Delivery of Nrf2 as therapy for protection against reactive oxygen species

Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to the subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.

AAV-mediated gene therapy for RPGR X-linked retinal degeneration

Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.

Hammerhead ribozymes that target components critical to HSV replication (ICP4, U20, U30, and U54) were synthesized and shown to efficiently cleave target RNA encoding a portion of these components in in vitro assays. Several cloned cell lines stably expressing these ribozymes were established. HSV-mediated plaque formation was dramatically reduced in cells stably transfected with an anti-HSV ribozyme compared to non-transfected cells.

Growing community of inventors

Gainesville, FL, United States of America

Alfred Samuel Lewin