Researchers Submit Patent Application, "Materials and Methods for Treatment of Hemoglobinopathies", for Approval (USPTO 20240226339).

Předmět:	PATENT applications GRANULOCYTE-colony stimulating factor RESEARCH personnel
Zdroj:	Stem Cell Week; 2024, p944-944, 1p
Abstrakt:	Researchers have submitted a patent application for a new method of treating genetic disorders that affect hemoglobin production and function. The method involves using genome engineering tools to permanently modify a gene called BCL11A, which is associated with these disorders. The researchers propose using this method to create treatments for conditions such as sickle cell anemia and thalassemia. The patent application was filed by Vertex Pharmaceuticals Incorporated and includes claims related to preparing ribonucleoproteins (RNPs) for gene editing. For more information, please refer to the full patent. [Extracted from the article]
Databáze:	Complementary Index
Externí odkaz:	Zobrazit plný text záznamu