A construct comprising a promoter specific for non-pigmented ciliary epithelial cells (NPCECs) is provided. In particular, the construct comprises a BEST2 minimal promoter that confers NPCEC-specific expression. The BEST2 minimal promoter may be operably linked to an expressible sequence such as a gene encoding a polypeptide of interest, a regulatory RNA sequence, a reporter gene, and the like. Such constructs may be provided in an expression vector, for example, with the BEST2 minimal promoter operably linked to an expressible sequence or in a host cell genetically modified with such an expression vector.

Provided are a fusion protein that improves gene editing efficiency and an application thereof. The fusion protein comprises a single-stranded DNA binding protein functional domain, nucleoside deaminase and nuclease. According to CBEs, when carrying our base conversion from C-G to T-A, nucleoside deaminase such as cytosine deaminase carries out deamination by using single-stranded DNA as a substrate, and by re-fusing the single-stranded DNA binding protein functional domain on the fusion protein of the nucleoside deaminase and nuclease, the chance of single-stranded DNA being exposed to the nucleoside deaminase is greatly increased, thereby significantly improving base editing efficiency. The present disclosure provides a breakthrough improvement of single-base gene editing technology and can greatly promote the application thereof in aspects such as gene editing, gene therapy, cell therapy, animal model making, and crop genetic breeding.

A method of treating human epidermal growth factor receptor 2 (HER2)-positive breast cancer in a subject in need thereof is provided, including administering to the subject an effective amount of a therapeutic agent that inhibits a nucleic acid that encodes FAK family-interacting protein of 200 kDa (FIP200). Also provided is a method of inhibiting metastasis of human epidermal growth factor receptor 2 (HER2)-positive breast cancer in a subject.

Disclosed is a combination of two or more lipase enzymes, and its use for treating a lipid digestion deficiency and/or a digestive disorder. At least one lipase enzyme has a pH optimum at an acidic pH value, while at least one other lipase enzyme has a pH optimum at an alkalic pH value.

Some embodiments provided herein relate to methods, systems and kits for providing consistent intracoronary administration of a biologic to subjects having diverse coronary anatomies. In some embodiments, the biologic is an adeno-associated virus serotype 1 (AAV1) vector encoding sarcoplasmic/endoplasmic reticulum ATPase 2a (SERCA2a) protein.

Some embodiments provided herein relate to methods, systems and kits for providing consistent intracoronary administration of a biologic to subjects having diverse coronary anatomies. In some embodiments, the biologic is an adeno-associated virus serotype 1 (AAV1) vector encoding sarcoplasmic/endoplasmic reticulum ATPase 2a (SERCA2a) protein.

Disclosed herein is an anti-adhesion kit comprised of: (i) a fibrinogen solution component comprising: fibrinogen at a concentration of about 5 to 25 mg/ml; and free calcium ions at a concentration ranging from 0.1 μM to 1 mM; and (ii) a thrombin component containing thrombin. Further disclosed is an anti-adhesion kit comprised of: (i) a fibrinogen solution component containing fibrinogen at a concentration of 8% to 25% of total protein by weight, and optionally free calcium ions at a concentration ranging from 0.1 μM to 1 mM; wherein a total protein concentration ranges from about 80 to 120 mg/ml; and (ii) a thrombin component containing thrombin. Methods of using the kits e.g., to provide anti-adhesion curable compositions are also disclosed.

The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).

The present disclosure provides methods and compositions useful for the prophylactic and therapeutic amelioration and treatment of infections caused by Gram-negative bacteria, including Pseudomonas aeruginosa. The disclosure further provides compositions and methods of incorporating and utilizing lysin polypeptides of the present disclosure for augmenting the efficacy of antibiotics generally suitable for the treatment of Gram-negative bacterial infection.

The present disclosure provides methods and compositions useful for the prophylactic and therapeutic amelioration and treatment of infections caused by Gram-negative bacteria, including Pseudomonas aeruginosa. The disclosure further provides compositions and methods of incorporating and utilizing lysin polypeptides of the present disclosure for augmenting the efficacy of antibiotics generally suitable for the treatment of Gram-negative bacterial infection.