This disclosure provides methods to modulate cardiac regeneration in a mammalian cardiac cell or progenitor, comprising contacting the mammalian cardiac cell with a DOT1L gene modulator.

Nucleic acid molecules and compositions, and methods using the same are provided herein for intraocular gene-based delivery and expression of two or more proneural bHLH transcription factors in the retina. The nucleic acid molecules, compositions and methods disclosed herein stimulate regeneration of retinal interneurons from retinal Mller glia (MG) and reprogram the MG into bipolar, amacrine, horizontal, and/or ganglion cells. Such methods and nucleic acid molecules are used for vision restoration and/or treatment of a range of ocular diseases involving retinal degeneration after injury, disease, or vison loss.

Described herein is the discovery that TDP-43 proteinopathies may be induced in adult or neonatal animals bearing on one chromosome a mutant TARDBP gene encoding a mutant TDP-43 protein that lacks a functional nuclear localization signal (NLS) or a mutant TARDBP gene encoding a mutant TDP-43 protein that lacks a prion-like domain (PLD) and on the other homologous chromosome a TARDBP gene comprising a conditional knockout mutation. Knockout of the TARDBP gene comprising the conditional knockout mutation, e.g., using Cre recombinase, during the neonatal stage, e.g., at P0-P10, or during adulthood, e.g., at about 5 months of age, results in the mice exhibiting neuromuscular phenotypes such as early lethality, paralysis, weight loss, etc. These animals exhibit hallmark symptoms of ALS and that may be used in testing candidate agents useful in treating TDP-43 proteinopathies.

Provided herein are compositions, methods, and uses involving antibodies that immunospecifically bind glycosylated forms of MUC16, a tethered mucin protein. Also provided herein are uses and methods for managing, treating, or preventing disorders, such as cancer.

The present disclosure is directed, in some embodiments, to compositions and methods for inducible modification of a cell genome.

A genetically modified non-human animal in which an auxin-inducible degron system controls degradation of a target protein in a living body includes: a chromosome containing a first nucleic acid that encodes a mutant TIR1 family protein having a mutation at an auxin-binding site and having affinity to an auxin analog.

Described is a polypeptide including the intracellular domain of CX3CL1 (CX3CL1-ICD), including an amino acid sequence that is at least 90% identical to SEQ ID NO: 1 and pharmaceutical compositions including the above-described polypeptide and a pharmaceutically acceptable excipient. Also included are nucleic acids expressing the polypeptides and expression cassettes and vectors comprising the nucleic acids. The polypeptides can be used in methods of treating a subject exhibiting a symptom of a neurodegenerative disease and/or diagnosed with a neurodegenerative disease and methods of treating a subject exhibiting a symptom of diabetes and/or diagnosed with diabetes comprising administering to the subject the pharmaceutical composition described herein.

A liquid suspension product comprising a recombinant adeno-associated virus (rAAV) having an AAV8 capsid which is suitable for intra-retinal injection is provided herein. Also provided herein are liquid suspensions containing these rAAV8.aVEGF and methods of using same for treatment of wet AMD and other ocular conditions.

An auxin-inducible degron system kit that controls degradation of a target protein in a non-plant-derived eukaryotic cell, the kit containing a first nucleic acid that encodes a mutant TIR1 family protein having a mutation at an auxin-binding site, an auxin analog that has an affinity to the mutant TIR1 family protein and a second nucleic acid that encodes a degradation tag containing at least a part of an Aux/IAA family protein and having an affinity to a complex of the mutant TIR1 family protein and the auxin analog.

Methods for inducing basal cell carcinoma (BCC) or BCC tumors, as well as an inducible non-human animal models of BCC, are defined herein. The methods and animal models comprise targeting Ptch1 and/or a tumor suppressor gene via conditional expression of one or more short hairpin RNAs (shRNAs) in the skin of said animals.