The present invention features a neural organoid that recapitulates in vitro most characteristics of the brain (e.g., human), and methods of using this neural organoid to study disease and to identify therapeutic agents for the treatment of neurological diseases and disorders.

Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.

The present disclosure provides methods for generating retinal cell clusters for use in cellular component transfer therapy, retinal cell clusters generated by such methods, and compositions comprising such retinal cell clusters. The present disclosure also provides uses of the retinal cell clusters and compositions comprising thereof for preventing and/or treating inherited retinal degenerative diseases.

Disclosed herein are kits comprising transcription factors for inducing a fibroblast cell into an induced embryonic neural progenitor cell. The induced embryonic neural progenitor cell is then capable of differentiating into an astrocyte, an oligodendrocyte or a neuron. Also disclosed are the uses of the kit as a platform for selecting a drug candidate to treat neurological diseases.

Described herein are methods and compositions related to promoting maturation of in vitro-differentiated cardiomyocytes and in vitro-differentiated neurons, and methods and compositions using the resulting cardiomyocytes and neurons.

The present invention provides a method of producing brain organoids.

In certain aspects, the present disclosure provides methods and materials for detection and characterization of retinal ganglion cells in a sample. In accordance with certain embodiments, the present disclosure provides systems and methods for detection of disease or diseased states related to retinal ganglion cells. In some forms the disclosure provides for a method for diagnosing a disease or diseased state related to retinal ganglion cells in a patient, the method comprising the step of detecting in a body fluid of the patient one or more markers associated with the disease or diseased state.

The invention relates to a neural tissue unit for use in implantation into the nervous system of a human or non-human mammal, wherein said neural tissue unit contains differentiated post-mitotic neuronal cells in an extracellular matrix, said unit being obtained from a cellular microcompartment comprising a hydrogel capsule surrounding the neural tissue unit, and said hydrogel capsule being at least partially removed before use of the neural tissue unit. The invention also relates to a process for preparing such a neural tissue unit.

Methods of producing human stem cells are disclosed for parthenogenetically activating human oocytes by manipulation of O.sub.2 tension, including manipulation of Ca.sup.2+ under high O.sub.2 tension and contacting oocytes with serine threonine kinase inhibitors under low O.sub.2 tension, isolating inner cell masses (ICMs) from the activated oocytes, and culturing the cells of the isolated ICMs under high O.sub.2 tension. Moreover, methods are described for the production of stems cells from activated oocytes in the absence of non-human animal products, including the use of human feeder cells/products for culturing ICM/stem cells. Stem cells produced by the disclosed methods are also described.

The presently disclosed subject matter provides for in vitro methods of inducing differentiation of human stem cells into neural crest, cranial placode or non-neuro ectoderm precursors, and cells generated by such methods. The presently disclosed subject matter also provides for uses of such cells for treating neurodegenerative and pituitary disorders.