Patent Application Titled "Direct Raav-Mediated In Vivo Gene Editing Of Hematopoietic Stem Cells" Published Online (USPTO 20240216534).

A patent application titled "Direct Raav-Mediated In Vivo Gene Editing Of Hematopoietic Stem Cells" has been published online by the US Patent and Trademark Office. The patent application, filed by inventors Terence Flotte and Allison Keeler-Klunk, is assigned to the University of Massachusetts. The application describes compositions and methods for gene editing in cells or subjects, specifically targeting hematopoietic stem cells for the treatment of hemoglobinopathies such as sickle cell disease. The disclosure includes details about the nucleic acids, adeno-associated viruses (AAVs), and gene products involved in the gene editing process. The patent application also mentions the use of guide RNAs (gRNAs) and RNA-guided nucleases (RGNs) in the method. [Extracted from the article]