C12N2750/14023

Compositions and methods for the targeting of rhodopsin

Provided herein are Class 2 Type V CRISPR:gNA systems comprising Class 2 Type V CRISPR polypeptides (e.g. CasX), guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a RHO gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the rhodopsin protein. Also provided are methods of using such systems to modify cells having such mutations and utility in methods of treatment of a subject with a RHO-related disease, such as retinitis pigmentosa.

COMPOSITIONS AND METHODS FOR THE TARGETING OF RHODOPSIN

Provided herein are Class 2 Type V CRISPR:gNA systems comprising Class 2 Type V CRISPR polypeptides (e.g. CasX), guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a RHO gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the rhodopsin protein. Also provided are methods of using such systems to modify cells having such mutations and utility in methods of treatment of a subject with a RHO-related disease, such as retinitis pigmentosa.

REGENERATION OF RETINAL GANGLION CELLS
20220347320 · 2022-11-03 ·

Provided herein are compositions and methods for regenerating retinal ganglion cells (RGCs) from retinal neuron cells by activating transcription factors such as one or more of Atoh7, Brn3B, Sox4, Sox11, or Ils1. The retinal neuron cells may be interneuron cells such as amacrine cells, horizontal cells, and bipolar cell. The regenerated RGCs can project axons into discrete subcortical brain regions and establish retina-brain connections. They can respond to visual stimulation and transmit electrical signals into the brain. Therefore, the regenerated RGCs can replace damaged or degenerated RGCs, thereby treating vision impairment or blindness. The methods are likewise applicable to degenerated, damaged, or aged RGCs to stimulate them to regrow functional axons, thereby rejuvenating these RGCs.

Anti-HER2 vaccine based upon AAV derived multimeric structures

The present invention relates to parvovirus mutated structural proteins comprising insertions of mimotopes of a HER2, compositions, multimeric structures, medicaments and vaccines comprising the same, nucleic acids, expression cassettes, constructs, vectors and cells comprising the nucleic acids, methods of preparing the structural proteins and methods of inducing a B-cell response or of treating a HER2-related disease.

OLIGONUCLEOTIDES AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION
20220195428 · 2022-06-23 ·

Disclosed are oligonucleotides, compositions, and methods that may be useful in the treatment of age-related macular degeneration (AMD). The treatment of age-regulated macular degeneration (AMD) may involve inhibiting an miR-33 target nucleic acid. For example, inhibition of an miR-33 target nucleic acid may be achieved using antisense oligonucleotides targeting an miR-33 target nucleic acid, interfering oligonucleotides targeting an miR-33 target nucleic acid, or recombinant AAV particles including a vector encoding an antisense oligonucleotide or interfering oligonucleotide targeting an miR-33 target nucleic acid.

Murine parvovirus and uses thereof

The present disclosure relates to a novel murine parvovirus, sequences encoded thereby, and applications therefor. In one embodiment the disclosure provides a method for detecting the presence of a parvovirus in a sample, comprising detecting one or more nucleic acids or polypeptides derived from the parvovirus, or antibodies against the parvovirus, in the sample. Also provided are vectors and host cells comprising sequences encoded by the parvovirus and related sequences. Also provided are animal models of kidney disease associated with infection by the parvovirus.

Methods and kits for quantifying the removal of mock virus particles from a purified solution
11754565 · 2023-09-12 · ·

The present invention relates to a method of quantifying the amount of Mock Virus Particles (MVP) removed from a solution as a result of processing that solution through a purification technique. This method involves the steps of adding MVP to a solution, processing the solution through a purification technique, quantifying the amount of MVP removed from the solution. The present invention also relates to a kit that can be used in conjunction with the method. This kit will comprise at least one stock solution of MVP and at least one quantification solution.

Coronavirus immunogenic compositions and uses thereof

Provided in the present disclosure are immunogenic compounds, pharmaceutical formulations thereof and their use for inducing a protective immune response against 2019 novel coronavirus (SARS-CoV-2) infection and variants in a mammal.

CORONAVIRUS IMMUNOGENIC COMPOSITIONS AND USES THEREOF

CORONAVIRUS IMMUNOGENIC COMPOSITIONS AND USES THEREOF Provided in the present disclosure are immunogenic compounds, pharmaceutical formulations thereof and their use for inducing a protective immune response against 2019 novel coronavirus (SARS-CoV-2) infection and variants in a mammal.

CORONAVIRUS IMMUNOGENIC COMPOSITIONS AND USES THEREOF

Provided in the present disclosure are immunogenic compounds, pharmaceutical formulations thereof and their use for inducing a protective immune response against 2019 novel coronavirus (SARS-CoV-2) infection and variants in a mammal.