Curing Classical Homocysteinuria with Pediatric in vitro engineered Liver Organoid Transplantation?

Abstract

Organoids are groups of cells grown in the laboratory that have self- organized into cell structures resembling those of organs. The term "organoid" means "organ-like." In many cases, the cells and cell structures give organoids abilities similar to those of the organs they resemble. Brain organoids, for example, develop layers of nerve cells (neurons) with signaling activity and even "brain regions" that resemble areas of the human brain. Currently, organoids created by researchers may bear a strong resemblance to a fully mature organ in certain respects, but crucial differences still exist. Intestinal organoids have a variety of cellular structures that resemble parts of the intestinal mucosa, but are typically the size of a pea and thus not nearly as large or complex as our intestinal tract. But even though they are small, or do not correspond one hundred percent to entire organs, scientists can learn a lot from organoids. Experts believe that organoids represent the "next generation" of biological tools for research, drug development and medicine. Liver Organoid engineering could play an important role in treating and curing classic homocysteinuria in childhood by resetting the disturbed function of cystathion beta synthetase activity in the implanted organoid liver. This manuscript focus on liver organoid research to date with special attention to classic homocysteinuria in childhood.