A transfection reagent composition comprising: 30-60 MOL. % of a cationic lipid, or a pharmaceutical acceptable salt thereof; 10-60 MOL % of a structural lipid; 10-20 MOL % of a triglyceride; and 0.1 to about 10 MOL. % of a stabilizing agent, is provided. The transfection agent is effective in transfecting cells, particularly neurons, with siRNA, mRNA and plasmid nucleic acid, Sand maintaining viability of the cells as well as activity of the delivered nucleic acid.

Methods for using gene expression changes and mutations in neural organoids to identify neural networks that predict the onset of Alzheimer's disease and associated comorbidities are disclosed.

Compositions and methods are provided for genetically modifying cells to guide in situ chemical synthesis of electroactive, conductive, or insulating polymers on plasma membranes, organelle membranes, or subcellular surfaces of cells. In particular, compositions and methods are provided for genetically modifying excitable cells such as neurons, muscle cells, and endocrine cells to guide in situ chemical synthesis of polymers on the extracellular side of the plasma membrane. The subject methods can be used in various applications, for example, to assemble polymers in vivo at targeted locations to modulate electrical conduction and create new electrical conduction pathways, allow cell-type-specific neuromodulation, provide a conductive structure on cells for connection to electrodes, sensors, or other external electronic and electrochemical devices, and create a durable structure to replace damaged tissue for use in regenerative medicine.

Methods of determining the suitability of a subject for treatment with a therapeutic agent are provided. Methods of providing a personalized treatment protocol based on suitability of a subject to be treated with a therapeutic agent are also provided, as are methods of treating those subjects who are suitable.

Methods are provided for the production of photoreceptor cells and photoreceptor progenitor cells from pluripotent stem cells. Additionally provided are compositions of photoreceptor cells and photoreceptor cells, as well as methods for the therapeutic use thereof. Exemplary methods may produce substantially pure cultures of photoreceptor cells and/or photoreceptor cells.

Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.

Progenitor cells were isolated, purified and expanded using a microfluidic based cell sorting approach. The methods were successfully in purifying cone progenitor cells (CPCs) defined based on a proliferative population expressing cone arrestin and Red/Green (R/G) opsin at greater than 80% using a two multistage approach.

Human raphe nuclei organoids or spheroids (hRNS) are generated in vitro, which may be generated at least in part from human pluripotent stem (hPS) cells. Such spheroids model the human raphe nuclei and comprise specific sets of cells, e.g. serotonergic neurons, that are associated with the raphe nuclei of a human, and can be assembled with cortical spheroids (hCS) to generate functional human neuromodulatory circuits.

A bioreactor device includes a solid substrate having a first face and a second face. The solid substrate at least partially defines a perfusion channel, a plurality of chambers, a fluidic inlet, and a fluidic outlet. A first sheet disposed over the first face and a second sheet disposed over the second face. Characteristically, the combination of the solid substrate, the first sheet and the second sheet define the perfusion channel and each chamber of the plurality of chambers. The plurality of chambers are arranged in rows of chambers in which adjacent chambers are positioned at opposite side of the perfusion channel. The perfusion channel extends from the fluidic inlet and the fluidic outlet having a serpentine path along each row of chambers with each chamber being in fluid communication with the perfusion channel.

Disclosed are methods for preparing retinal pigment epithelium (RPE) cells from pluripotent stem cells (PSCs). More particularly, it represents an automated method that combines in a sequential manner three differentiating agents to direct the differentiation of human PSCs into RPE cells.