Gene therapy may be possible cure for Hurler syndrome- Mouse Study

According to the research carried out by the Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) explains unique strategy for treating the Hurler Syndrome. The report published in the Proceedings of the National Academy of Sciences (PNAS) explains that the used blood platelets and the bone marrow cells can be gene therapy to mouse models of the human genetic disorder Hurler syndrome. This is a fatal condition that can cause organ damage along with other medical complications. One of the authors, Dao Pan, PhD, researcher in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's explains, “Our findings demonstrate a unique and somewhat surprising delivery pathway for lysosomal enzymes. We show proof of concept that platelets and megakaryocytes are capable of generating and storing fully functional lysosomal enzymes, which can lead to their targeted and efficient delivery to vital tissues where they are needed.”