Abstract: The present invention relates to an expression cassette allowing expression of a functional LRIT3 protein in mammal eyes; said expression cassette is inserted in an expression vector, preferably an adeno-associated virus (AAV); accordingly, the present invention further relates to a recombinant adeno-associated virus (AAV) vector carrying a nucleic acid sequence encoding a normal LRIT3 gene, or fragment thereof, under the control of regulatory sequences which express the product of the gene in the ocular cells, a pharmaceutically acceptable composition comprising such a recombinant AAV vector and to its use for the treatment of congenital stationary night blindness

Abstract: An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3?UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3?UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.

Abstract: The present invention relates to methods and composition for use in the treatment of retinal degeneration, in particular retinal degeneration due to retinal pigment epithelium dysfunction.

Abstract: The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated-K+ channel (GIRK), in particular GIRK2, activated by G proteins recruited by cone opsin expressed in degenerating cones for use in the treatment of patients with RCD.

Abstract: Devices and methods for reversibly stimulating neuronal cells in a subject. The devices include a module for generating ultrasounds at 4 MHz or more to stimulate neuronal cells expressing mechanosensitive channels with the ultrasounds. The methods include expressing mechanosensitive channels into neuronal cells and exposing the cells to ultrasounds at 4 MHz or more. Also, the use of the methods or devices for visual restoration in a subject.

Abstract: Several new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea are provided. The delivery modality relies on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector comprising a VP1 capsid protein as set forth in SEQ ID NO: 11 and the polynucleotide of interest under the control of the pR1.7 promoter as set forth in SEQ ID NO: 12 are also provided.

Abstract: Disclosed is a method utilizing (R)-2-(2-aminothiazol-4-yl)-4?-[2-[(2-hydroxy-2-phenylethyl)amino]ethyl]acetic acid anilide or an analogue, pharmaceutically acceptable salt or solvate thereof for the treatment of a retinal disease, more specifically using mirabegron for the treatment of age-related macular degeneration. Also Disclosed is a pharmaceutical composition, a medicament and a kit for the treatment of age-related macular degeneration.

Abstract: An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3?UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3?UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.

Abstract: An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3?UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3?UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.

Abstract: An insulin sensitizer for use in the prevention and/or treatment of the damages caused by retinal detachment, and in particular for preventing or treating the loss of vision induced by retinal detachment. Also, a composition including the insulin sensitizer for use in the prevention and/or treatment of the damages caused by retinal detachment and a device including the composition.

Abstract: The present invention concerns a new gene therapy approach to increase light-sensitivity in degenerating cones in advanced stages of rod-cone dystrophy (RCD) mediated by G-protein-gated-K+ channel (GIRK), in particular GIRK2, activated by G proteins recruited by cone opsin expressed in degenerating cones.

Abstract: Disclosed is chemical compounds C that are derivatives of norbixin and have tropism for the eye, and are intended to be used in the treatment of eye diseases in mammals, in particular in the context of altering the retinal pigment epithelium and more particularly in the context of age-related macular degeneration (AMD) and Stargardt's disease.

Abstract: Disclosed herein are methods of treating age-related retinal diseases by administering to the subject a therapeutically effective amount of a purine nucleoside phosphorylase (PNPase) inhibitor and/or a PNPase purine nucleoside substrate. In some examples, the subject can have AMD or glaucoma.

Abstract: The present invention concerns a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a compound selected in the group comprising (i) a polypeptide comprising an amino acid sequence selected in the group comprising the amino acid sequence of the long isoform in Homo sapiens of the RdCVF2 gene (SEQ ID NO: 10), orthologs, derivatives and fragments thereof, (ii) a polynucleotide coding for said polypeptide, (iii) a vector comprising said polynucleotide, and (iv) a host cell genetically engineered expressing said polypeptide; the use of such a composition for the manufacture of a medicament for treating and/or preventing a neurodegenerative disorder in a subject; and a method of testing a subject thought to have or be predisposed to having a neurodegenerative disorder.

Abstract: An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3?UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3?UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.

Abstract: The present invention concerns a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a compound selected in the group comprising (i) a polypeptide comprising an amino acid sequence selected in the group comprising the amino acid sequence of the long isoform in Homo sapiens of the RdCVF2 gene (SEQ ID NO: 10), orthologs, derivatives and fragments thereof, (ii) a polynucleotide coding for said polypeptide, (iii) a vector comprising said polynucleotide, and (iv) a host cell genetically engineered expressing said polypeptide; the use of such a composition for the manufacture of a medicament for treating and/or preventing a neurodegenerative disorder in a subject; and a method of testing a subject thought to have or be predisposed to having a neurodegenerative disorder.

Abstract: Disclosed herein are embodiments of a polymer-based implant and methods of making and using the same. The polymer-based implant comprises a polymer component and a therapeutic agent. In some embodiments, the polymer-based implant can be used to treat and/or prevent retinal diseases and/or retinopathies. The polymer-based implant exhibits physical properties that provide the ability to safely place the polymer-based implant in an ocular region without undesired diffusion and also to allow for controlled and timely release of the therapeutic agent to a desired region of the ocular region, such as the retina. In particular disclosed embodiments, the polymer-based implant can be used for safe and effective gene therapy.

Abstract: The present invention relates to a nucleic acid sequence derived from the regulatory region of the human gamma-synuclein gene and having a promoter activity in retinal ganglion cells. The present invention also relates to expression cassettes or vectors comprising said promoter operably linked to a nucleic acid sequence encoding a polypeptide of interest as well as viral particles or host cells comprising said expression cassette or vector. The present invention also relates to the use of said expression cassettes, vectors, viral particles or cells in the treatment of ocular disease, in particular ocular disease associated with retinal ganglion cell or photoreceptor cell degeneration.

Abstract: The invention relates to compounds which activate the BASIGIN signalling pathway, preferably agonists of BASIGIN, for the treatment of neurodegenerative disorders.

Abstract: The present invention concerns a neuroglobin agonist for use in the treatment or prevention of a mitochondrial disease associated with respiratory chain complex I (RCCI) deficiency and/or respiratory chain complex III (RCCIII) deficiency.