Described herein are compositions and methods for modulating cellular senescence of a cell or induction of the senescence-associated secretory phenotype (SASP) in a cell. The methods generally comprise modulating the level or activity of IRE1a as a mean to control cellular senescence and induction of the SASP. Also described are methods for treating and preventing ocular vascular diseases comprising contacting cells in an eye of a subject with a biguanide compound and ophthalmic compositions comprising a biguanide compound.

The disclosure provides bifunctional tag constructs and related methods useful for transporting macrobiomolecules, such as nucleic acids and proteins, across plasma membranes. The bifunctional tags comprise an affinity domain conjugated to hydrophobic domain. The affinity domain is configured to noncovalently bind to the macrobiomolecule, whereas the hydrophobic domain is configured to interact with the plasma membrane. In certain embodiments, the plurality of bifunctional tags can noncovalently associate along the length of a macrobiomolecule, thus increasing the interaction with and penetration through the plasma membrane.

A recombination parvovirus vector that comprises a parvovirus capsid and a double-stranded vector genome having a sense-strand and an antisense-strand. The sense-strand comprises in the 5′ to 3′ direction: a parvovirus terminal repeat at the 5′ end a coding sequence of a gene of interest (GOI); and a parvovirus terminal repeat at the 3′ end. The vector genome further comprises a RNA destabilization/destruction domain (RDDD).

This disclosure relates to novel HTT-1A targeting sequences. Novel HTT-1A targeting oligonucleotides for the treatment of neurodegenerative diseases are also provided.

The present disclosure provides a combination of antisense RNA sequences and use thereof in the production of abortive tilapia, belonging to the technical field of molecular biology and reproductive biology, the combination of antisense RNA sequences includes antisense RNA of steroidgenic factors SF1-1 and SF1-2; the nucleotide sequences of Anti-SF1-1-I, Anti-SF1-1-II, Anti-SF1-2-I and Anti-SF1-2-II are set forth in SEQ ID NO. 1-SEQ ID NO. 4 respectively. The method of the present disclosure introduces antisense RNA fragments into the eggs through the fertilization hole to realize effective and accurate targeted intervention for regulating the gene expression, and the method has the advantages of simple operation, minimal egg damage, high success rate, stable phenotype after breeding, and excellent application prospects.

The invention relates to polynucleotide agents targeting a Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such polynucleotide agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.

The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5′-GUGGCUAACAGAAGCU (SEQ ID NO:1), 5′-GGGAACAUGCUAAAUAC (SEQ ID NO:2), 5′-AGACACAAAUUCCUGAGA (SEQ ID NO:3), or 5′-CUGUUGAGAAA (SEQ ID NO. 4), and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.

Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

The present invention relates to a novel antisense transcript to the human SLC2A1 (Glut1) gene, variants and fragments thereof. This antisense transcript can be used to modulate Glut1 expression and serve to restore Glut1, and as a therapeutic for treating or preventing Glut 1 deficiency syndrome, or other Glut1 related conditions including certain cancers, diabetes, Alzheimer's disease, and retinitis pigmentosa.

Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.