Provided are methods of making and using animal models for brain inflammation and white matter degeneration.

Methods are provided for delivery of at least one agent into egg(s) from an egg-producing aquatic animal including contacting fertilized or unfertilized egg(s) from said egg-producing aquatic animal with the at least one agent in the presence of a guanidine-containing compound capable of enhancing the permeability of the chorion of the egg(s). Methods are provided for the drug screening and compound toxicity assays. Methods are also provided for the production of reproductively sterile fish and aquatic animals for aquaculture, the aquarium trade, and control of invasive species are described. The methods include disruption of gonadal development through the administration of agents that lead to the failure of fertile gonadal development. Agents may be delivered to the eggs directly before the fertilization or post fertilization by contacting eggs in an immersion medium including the agent of interest.

Methods for the production of reproductively sterile fish and aquatic animals for aquaculture, the aquarium trade, and control of invasive species are described. The methods include disruption of gonadal development through the administration of compounds that lead to the failure of fertile gonadal development. Compounds may be delivered to the eggs prior to fertilization or water activation or post fertilization and water activation by contacting unfertilized or pre-water-activated fertilized eggs or fertilized eggs in an immersion medium including the compound of interest. Compounds may be conjugated with a molecular transporter compound effective for chorionic transport of the conjugate. The compounds may be antisense Morpholino oligomers that are capable of effectively suppressing the expression of the dead end gene or other essential genes for germ cell development in fish and other egg-producing aquatic animals.

A method of modulating some or all copies of a gene in a cell is provided including introducing into a cell one or more ribonucleic acid (RNA) sequences that comprise a portion that is complementary to all or a portion of each of the one or more target nucleic acid sequences, and a nucleic acid sequence that encodes a Cas protein and maintaining the cells under conditions in which the Cas protein is expressed and the Cas protein binds and modulates the one or more target nucleic acid sequences in the cell.

Aspects of the invention described herein relate to synthetic, modified RNAs and their use in vivo to modulate gene expression. Aspects of the invention further relate to the use of these synthetic, modified RNAs in myocytes, cardiomyoctes, and tumors.

Provided herein are compositions, methods, and devices for the treatment and prevention of atrial fibrillation (AF) using gene therapy techniques. In particular, oxidative stress (OS) and parasympathetic nervous system signaling are inhibited to prevent and/or reverse the electrical remodeling that underlies AF.

Described herein are block copolymers, and methods of making and utilizing such copolymers. The described block copolymers are disruptive of a cellular membrane, including an extracellular membrane, an intracellular membrane, a vesicle, an organelle, an endosome, a liposome, or a red blood cell. Preferably, in certain instances, the block copolymer disrupts the membrane and enters the intracellular environment. In specific examples, the block copolymer is endosomolytic and capable of delivering an oligonucleotide (e.g., an mRNA) to a cell. Compositions comprising a block copolymer and an oligonucleotide (e.g., an mRNA) are also disclosed.

Provided are diagnosis and treatment methods of autism spectrum disorder, based on an activity regulation mechanism of quiescent neural stem cells, and use thereof.

The present invention provides a composition which comprises the following Thyroid Stimulating Hormone Recept or (TSHR) peptides: (i) all or part of the amino acid sequence KKKKYVSIDVTLQQLESHKKK (SEQ ID NO: 1), or a part thereof, or a sequence having at least 60% sequence identity to SEQ ID NO:1; and (ii) all or part of the amino acid sequence GLKMFPDLTKVYSTD (SEQ ID NO: 2), or a part thereof, or a sequence having at least 60% sequence identity to SEQ ID NO:2. The present invention also relates to the use of such a composition for the prevention or suppression of activating autoantibody formation in Graves' disease.

The present invention is directed generally to eukaryotic cells comprising single-celled organisms that are introduced into the eukaryotic cell through human intervention and which transfer to daughter cells of the eukaryotic cell through at least five cell divisions, and methods of introducing such single-celled organisms into eukaryotic cells. The invention also provides methods of using such eukaryotic cells. The invention further provides single-celled organisms that introduce a phenotype to eukaryotic cells that is maintained in daughter cells. The invention additionally provides eukaryotic cells containing magnetotactic bacteria.