Methods for the treatment and prevention of laminopathies and diseases characterised by hyperlipidemia through LING complex inhibition are disclosed. In particular, LING complex disruption by expression of dominant-negative LING complex proteins alleviates pathophysiology in Lmna mutation-associated muscular dystrophy, progeria, and dilated cardiomyopathy. In addition, LING complex disruption by expression of dominant-negative LING complex proteins also alleviates pathophysiology in mouse models of atherosclerosis and familial hypercholesterolemia.

Immunogenic peptide fragments of metalloprotease ADAMTS-7 including a first short peptide, which is any one of the followings: a short peptide having the amino acid sequence shown in SEQ ID NO: 1 in the sequence listing; a short peptide having the amino acid sequence shown in SEQ ID NO: 2 in the sequence listing; a short peptide having the amino acid sequence shown in SEQ ID NO: 3 in the sequence listing; a short peptide having the amino acid sequence shown in SEQ ID NO: 4 in the sequence listing. The description includes uses of conjugates containing the above short peptides and vaccines containing the conjugates. The vaccines containing the short peptides can remarkably inhibit the intimal neogenesis in the vascular restenosis mouse models and the occurrence of atherosclerosis in high-fat-fed mice, and can be used for the prevention or treatment of atherosclerosis and/or vascular restenosis.

The present disclosure provides methods and compositions for high frequency mouse transgenesis using, for example, a Bxb1 landing pad.

The present invention relates to compositions, methods, uses and kits for the treatment of renal cystogenesis. In particular, the compositions, methods, uses and kits are particularly useful, but not limited to, the treatment or prevention of Polycystic Kidney Disease. In one aspect, the prevent invention provides a method of minimising or delaying renal cystogenesis in a subject in need thereof, the method comprising inhibiting AKT in the subject, or reducing the level of Aurora kinase in the subject, thereby minimising or delaying renal cystogenesis.

Described herein are compositions comprising AAV vectors comprising a sequence encoding mucolipin 1, and methods of use thereof for gene therapy of Mucolipidosis IV (MLIV).

Described herein are compositions and methods for treating a subject having or at risk of developing Crohn's disease. Using the compositions and methods of the disclosure, a patient, such as an adult human patient, may be provided one or more agents that elevate the expression and/or activity levels of Nucleotide-binding oligomerization domain-containing protein 2 (NOD2). Exemplary agents that may be used in conjunction with the compositions and methods of the disclosure for this purpose include cells, such as pluripotent cells, that express NOD2.

Disclosed herein are novel variants of KCVN2 and their use, for example, in methods of treating a subject with a retinal disorder, such as CDSRR.

A system for treating cancer and evaluating chemo-preventive potential of PHC and its prepared chitosan nanoparticles is described. The rats are divided into eight groups, from which group 1 is served as normal control, and group 2-8 are given single dose of DEN and repeated dose of CCl.sub.4, wherein freshly prepared solution of DEN in normal saline is used for the induction of HCC in rats by administering 200 mg/kg, i.p., PHC (2:1:1) in normal saline suspension to administer at doses of 900 mg/kg, wherein serum and tissue samples are collected after anesthetizing overnight fasted rats using intraperitoneal administration of thiopentone sodium at a dose of 40 mg/kg, wherein the collected serum and tissue samples is treated and thereby the chemo-preventive potential of PHC (2:1:1) and its prepared chitosan nanoparticles is evaluated upon determining liver markers, antioxidant parameters, total bilirubin, protein, lipid peroxidation, and liver cancer biomarkers.

Genetically modified non-human animals comprising a humanized interleukin-15 (IL-15) gene. Cells, embryos, and non-human animals comprising a human IL-15 gene. Rodents that express humanized or human IL-15 protein.

Genetically modified mice and methods and compositions for making and using the same are provided, wherein the genetic modification comprises humanization of an FcγRI protein.