C12Y301/26004

Compounds and Methods for the Modulation of Proteins

In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.

METHODS FOR VARIANT DETECTION

The invention can be used to provide a more efficient and less error-prone method of detecting variants in DNA, such as SNPs and indels. The invention also provides a method for performing inexpensive multiplex assays.

METHODS FOR VARIANT DETECTION

The invention can be used to provide a more efficient and less error-prone method of detecting variants in DNA, such as SNPs and indels. The invention also provides a method for performing inexpensive multiplex assays. The invention also provides methods for detection of DNA sequences altered after cleavage by a targetable endonuclease, such as the CRISPR Cas9 protein from the bacterium Streptococcus pyogenes.

Synthetic reverse transcriptases and uses thereof
11046940 · 2021-06-29 · ·

The present disclosure provides non-natural reverse transcriptases for conducting reverse transcription. The non-natural reverse transcriptases herein may have increased thermostability and can conduct reverse transcription more efficiently than natural reverse transcriptases.

OLIGONUCLEOTIDES FOR MODULATING GSK3B EXPRESSION

The present invention relates to antisense oligonucleotides that are capable of reducing expression of GSK3B in a target cell. The antisense oligonucleotides hybridize to GSK3B pre-mRNA. The present invention further relates to conjugates of the antisense oligonucleotide, pharmaceutical salts and pharmaceutical compositions and methods for treatment or alleviation of conditions such as cancer, inflammatory diseases, neurological diseases, neurological injury, neuronal degeneration, psychiatric diseases and Type 2 diabetes.

OLIGONUCLEOTIDES FOR MODULATING TAU EXPRESSION

The present invention relates to antisense oligonucleotides that are capable of modulating expression of Tau in a target cell. The oligonucleotides hybridize to MAPT mRNA. The present invention further relates to conjugates of the oligonucleotide and pharmaceutical compositions and methods for treatment of Tauopathies, Alzheimzer's disease, fronto-temporal dementia (FTD), FTDP-17, progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), corticobasal ganglionic degeneration (CBD), epilepsy, Dravet syndrome, depression, seizure disorders and movement disorders.

RNase H mutants in an emulsion

The invention is directed to methods and kits for performing an RNase H2-mediated cleavage reaction on a sample in an emulsion.

Methods for variant detection

The invention can be used to provide a more efficient and less error-prone method of detecting variants in DNA, such as SNPs and indels. The invention also provides a method for performing inexpensive multiplex assays. The invention also provides methods for detection of DNA sequences altered after cleavage by a targetable endonuclease, such as the CRISPR Cas9 protein from the bacterium Streptococcus pyogenes.

Compounds and methods for the modulation of proteins

In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.

Polynucleotide Based Movement, Kits and Methods Related Thereto
20200332350 · 2020-10-22 ·

This disclosure relates DNA based movement of objects. In certain embodiments, particles, pairs of particles, or rods are conjugated with single stranded DNA that hybridizes to a single stranded RNA that is conjugated to a substrate. When the DNA particle, pair of particles, or rod interacts with the surface RNA in the presence of an endonuclease, such as RNase H and the DNA hybridizes to the RNA, then the particle, pair of particles, or rod moves along the surface. The complementarity of the DNA and RNA affect the velocity.