Abstract: The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina, for the treatment of retinal disorders and diseases.

Abstract: The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.

Abstract: The present disclosure provides compositions and methods for the treatment of ocular diseases associated with angiogenesis, particularly wet age-related macular degeneration.

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding hum n alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

Abstract: The invention relates generally to dimeric molecular complexes comprising two fusion proteins. Each fusion protein comprises a biological effector moiety, a polypeptide spacer sequence, and an IgE CH4 dimerization domain. The dimeric molecular complexes may be conjugated, at a defined site, to other molecules including drug moieties, cytotoxic agents, labels (such as detectable labels), or biocompatible polymers.

Abstract: The present invention provides an improved, expression system for the regulated expression of an encoded protein or nucleic acid therapeutic molecule in the cells of a subject, for use in the treatment of disease. In particular, the present invention provides an improved, regulated gene expression system, and pharmaceutical compositions and uses thereof for treatment of disease.

Abstract: The present invention provides an improved, expression system for the regulated expression of an encoded protein or nucleic acid therapeutic molecule in the cells of a subject, for use in the treatment of disease. In particular, the present invention provides an improved, regulated gene expression system, and pharmaceutical compositions and uses thereof for treatment of disease.

Abstract: The present invention provides an improved, expression system for the regulated expression of an encoded protein or nucleic acid therapeutic molecule in the cells of a subject, for use in the treatment of disease. In particular, the present invention provides an improved, regulated gene expression system, and pharmaceutical compositions and uses thereof for treatment of disease.

Abstract: The present invention provides an improved, expression system for the regulated expression of an encoded protein or nucleic acid therapeutic molecule in the cells of a subject, for use in the treatment of disease. In particular, the present invention provides an improved, regulated gene expression system, and pharmaceutical compositions and uses thereof for treatment of disease.

Abstract: The present invention provides an improved, expression system for the regulated expression of an encoded protein or nucleic acid therapeutic molecule in the cells of a subject, for use in the treatment of disease. In particular, the present invention provides an improved, regulated gene expression system, and pharmaceutical compositions and uses thereof for treatment of disease.

Abstract: This system is for use by a visitor to a facility that has multiple occupants, public-subscriber or other phone service, and an individual phone outlet at each occupant's part of the facility. A microphone and speaker are provided so that a visitor seeking access can speak with an occupant. A communication circuit carries signals between (1) the mike and speaker and (2) the phone service--to enable the conversation through an individual phone outlet. Manual controls, preferably including a standard phone keypad, are positioned for use by the visitor. An electronic memory holds directory information, particularly including occupant names and corresponding dial-up codes. An LCD, behind a protective window, shows the visitor the directory data. Names and other directory data can be fed into the memory remotely, preferably through either: ordinary phone connections using standard DTMF signals--with automated confirmation of input data by a voice-synthesis chip--or a modem.

Abstract: A liquid-crystal display, preferably twenty-five lines of eighty characters each, is mounted in a case. The temperature of the display medium is maintained within operating limits for the display--notwithstanding ambient temperature and humidity variations over generally normal ranges for at least the temperate zones, and even if the display unit is placed to receive direct sunlight. The unit works outdoors as well as in buildings that lack temperature control. This is accomplished preferably by circulating air from outside the case through a space between the display face and an unperforated protective polycarbonate window. Intake and exhaust plena are long and narrow enough to deter manual access to the display and thus van dalism; however, the plena are straight and sufficiently free of obstructions that the fan can be a very small, low-power, quiet unit. A temperature sensor controls the fan, and also the contrast-adjusting voltage of the display --based on data in a digital-memory "look up" table.

Abstract: A liquid-crystal display, preferably twenty-five lines of eighty characters each, is mounted in a case. The temperature of the display medium is maintained within operating limits for the display--notwithstanding ambient temperature and humidity variations over generally normal ranges for at least the temperate zones, and even if the display unit is placed to receive direct sunlight. The unit works outdoors as well as in buildings that lack temperature control. This is accomplished preferably by circulating air from outside the case through a space between the display face and an unperforated protective polycarbonate window. Intake and exhaust plena are long and narrow enough to deter access to the display and thus vandalism; however, the plena are straight and sufficiently free of obstructions that the fan can be a very small, low-power, quiet unit. A temperature sensor controls the fan, and also the contrast-adjusting voltage of the display--based on data in a digital-memory "look up" table.