C12N2820/007

PROMOTERS FOR SPECIFIC EXPRESSION OF GENES IN CONE PHOTORECEPTORS

Disclosed herein are synthetic cone-specific promoters. The promoters can be used in nucleic acids, vectors, or host cells to drive expression of a heterologous polypeptide or RNA in retinal cells, particularly cones.

ENGINEERED PHOSPHOENOLPYRUVATE CARBOXYLASE ENZYMES

The present disclosure provides plants that express a variant phosphoenolpyruvate carboxylase (PEPC) enzyme. The plants have enhanced resistance to aluminum than comparable plants that lack the variant PEPC enzyme. In addition, the plants more effectively sequester carbon, extract phosphate, and produce oxaloacetate-derived amino acids and glucose than comparable plants that lack the variant PEPC enzyme. The disclosure also provides tools for production of plants that express the variant PEPC enzyme.

Recombinant Viral Genomes and Related Compositions and Methods
20250235493 · 2025-07-24 ·

Provided are recombinant viral genomes. The viral genomes find use in a variety of contexts including for the production of viruses effective in inducing death of cells exhibiting a signal specific to a disease or disorder in a subject, where the viral life cycle is dependent upon the signal specific to the disease or disorder. According to some embodiments, the viral genomes comprise one or more transcription units comprising a sequence encoding a first fusion protein comprising a protease and a first binding domain that binds to a first target, and a sequence encoding a second fusion protein comprising a membrane-targeting signal, a second binding domain that binds to a second target, a cleavable substrate for the protease, and either a transcriptional effector protein or viral protein encoded by a viral gene deleted in the viral genome. Also provided are related cells, virions, pharmaceutical compositions and methods of use.

MEASUREMENT OF SOMATIC L1 RETROTRANSPOSITION ACTIVITY

The invention relates to an expression vector operable in vertebrate liver cells, having an expression cassette with a bidirectional promoter, driving operably linked protein expression by a first side and a second side, a first expression unit, under the control of the first side of the promoter, said first expression unit comprising a positive selectable marker gene, a second expression unit, under the control of the second side of the promoter, comprising an ORFeus reporter element wherein said ORFeus reporter element comprises a gene encoding L1-ORF land a retrotransposition reporter gene encoding a retrotransposition reporter protein, wherein preferably the retrotransposition reporter protein from said retrotransposition reporter gene is provided only when the ORFeus reporter element is subject to retrotransposition. The invention also relates to transgenic animals which are useful to detect somatic retrotransposition.

Single-vector Type I vectors

The invention relates to the production and use of Cas-encoding sequences and vectors comprising these. Aspects of the invention provide products, vectors, delivery vehicles, uses and methods for producing Cas-encoding sequences in bacterial or archaeal cells.

GENE DELIVERY TARGETING NEURAL STEM CELLS AND NEURAL PROGENITOR CELLS

Methods of expressing a heterologous nucleic acid in neural stem and progenitor cells (NSPCs), comprising transducing NPSCs with an adeno-associated virus 6 (AAV6) vector comprising the heterologous nucleic acid are provided. Methods of treating a neurological disorder in a mammalian subject, comprising administering to the subject a therapeutically effective amount of an adeno-associated virus 6 (AAV6) vector comprising a heterologous nucleic acid molecule, such as Gsx1, are also provided.

Solving Brain Circuit Function and Dysfunction With Computational Modeling and Optogenetic Functional Magnetic Resonance Imaging
20250325707 · 2025-10-23 ·

Methods, systems, and devices, including computer programs encoded on a computer storage medium are provided for optimizing neurostimulation therapy for treatment of neurological and neurodegenerative diseases. Joint dynamic causal modeling and biophysics modeling are used for optimization of the stimulation targets and parameters. In particular, methods of performing neuromodulation to suppress b-band oscillations in the brain of a subject are provided.

SINGLE-VECTOR TYPE I VECTORS

The invention relates to the production and use of Cas-encoding sequences and vectors comprising these. Aspects of the invention provide products, vectors, delivery vehicles, uses and methods for producing Cas-encoding sequences in bacterial or archaeal cells.