Patent classifications
C12N2310/141
Nitrogen responsive transcription factors in plants
Methods for increasing or decreasing Nitrogen (N) uptake/assimilation and/or usage in plants comprising over-expressing or repressing one or more transcription factors that have been identified by evaluating temoporal transcription of the TFs in response to N signaling and validated based on TF perturbation studies in plant cells and plants. Combinations of TFs may be used, where each TF may be independently induced or repressed to achieve a desired increase or decrease in N uptake/assimilation.
Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
Kit, device and method for detecting prostate cancer
This invention provides a kit or device for detection of prostate cancer and a method for detecting prostate cancer. This invention provides a kit or device for detection of prostate cancer comprising a nucleic acid capable of specifically binding to an miRNA in a sample from a subject or a complementary strand thereof and a method for detecting prostate cancer comprising measuring the miRNA in vitro.
Kit, device and method for detecting prostate cancer
This invention provides a kit or device for detection of prostate cancer and a method for detecting prostate cancer. This invention provides a kit or device for detection of prostate cancer comprising a nucleic acid capable of specifically binding to an miRNA in a sample from a subject or a complementary strand thereof and a method for detecting prostate cancer comprising measuring the miRNA in vitro.
Method for Treatment of Alzheimer's Disease
The presently-disclosed subject matter generally relates to methods for treating a subject with Alzheimer's Disease, microhemorrhages, and neurological deficits. The presently-disclosed subject matter also relates to methods for treating a subject with Alzheimer's Disease, microhemorrhages, and neurological deficits with a composition that increases epoxyeicosatrienoic acids. The presently-disclosed subject matter further relates to a method of treating or preventing Alzheimer's Disease comprising administering an agent that increases vascular LRP1 expression.
OPTIDICER CONSTRUCT FOR AGE-RELATED MACULAR DEGENERATION
Provided are nucleotide sequences encoding polypeptides with ribonuclease III activity, wherein the nucleotide sequences have been modified to reduce their regulation by miRNAs. In some embodiments, the nucleotide sequences are at least 50% and as much as 100% identical to SEQ ID NO: 20 or SEQ ID NO: 22, and/or encode polypeptides that are at least 90% percent identical to SEQ ID NO: 23. Also provided are vectors and host cells that include the nucleotide sequences, methods for expressing the nucleotide sequences in cells, tissues, and organs, which in some embodiments can be in the eye of a subject in need thereof, methods for preventing and/or treating development of diseases or disorders and/or for restoring undesirably low DICER1 expression using the nucleotide sequences, and pharmaceutical compositions that have the presently disclosed nucleotide sequences.
OPTIDICER CONSTRUCT FOR AGE-RELATED MACULAR DEGENERATION
Provided are nucleotide sequences encoding polypeptides with ribonuclease III activity, wherein the nucleotide sequences have been modified to reduce their regulation by miRNAs. In some embodiments, the nucleotide sequences are at least 50% and as much as 100% identical to SEQ ID NO: 20 or SEQ ID NO: 22, and/or encode polypeptides that are at least 90% percent identical to SEQ ID NO: 23. Also provided are vectors and host cells that include the nucleotide sequences, methods for expressing the nucleotide sequences in cells, tissues, and organs, which in some embodiments can be in the eye of a subject in need thereof, methods for preventing and/or treating development of diseases or disorders and/or for restoring undesirably low DICER1 expression using the nucleotide sequences, and pharmaceutical compositions that have the presently disclosed nucleotide sequences.
ENGINEERED CIRCULAR RNA CIRCMIR-29B AND USE THEREOF IN PREPARATION OF MEDICINE FOR TREATING MUSCLE ATROPHY
The present disclosure belongs to the technical field of biomedicine, and provides an engineered circular RNA circmiR-29b and use thereof in preparation of a medicine for treating muscle atrophy. The present disclosure provides a circular RNA circmiR-29b including an effective sequence and a random sequence, wherein 6-13 repetitions of the effective sequence are connected in series, the random sequence is inserted between the effective sequences, and the nucleotide sequence of the effective sequence is shown in SEQ ID NO: 1. The present disclosure delivers circmiR-29b to skeletal muscle by AAV8, enabling stable expression of circmiR-29b in the skeletal muscle, thereby effectively inhibiting various types of muscle atrophy. Therefore, the present disclosure also provides a gene therapy based on the AAV8 virus vector delivering the engineered circular RNA circmiR-29b to achieve the objective of treating muscle atrophy.
Gene editing for hemophilia A with improved factor VIII expression
Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo. Also provided herein, in some embodiments, are materials and methods for knocking in a coding sequence encoding a synthetic FVIII having a B domain substitute into a genome.
Composition for regulating production of ribonucleic acid
The present disclosure relates to one or more compositions for increasing production of micro-RNA associated with decreasing production of a target biomolecule.