(Atlanta, December 9, 2017) — In four studies being presented today during the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, researchers announce promising findings on innovative tools and therapies for hard-to-treat blood disorders including sickle cell disease (SCD), beta thalassemia, and X-linked severe combined immunodeficiency (X-SCID). The studies employ unique therapeutic approaches and advanced engineering technologies such as the gene-editing platform CRISPR/Cas9, reflecting the fruits of many years of research dedication and innovation. “Achievements like these represent an enormous amount of determination on the part of physician-scientists to create treatments and cures for these patients,” said press briefing moderator Jonathan G. Hoggatt, PhD, principal faculty member of the Harvard Stem Cell Institute and Assistant Professor of Medicine at Harvard Medical School. “Making these tools and treatments a reality doesn’t happen overnight or in five years; it requires a great deal of perseverance both in the lab and in the clinic.” This press conference will take place on Saturday, December 9, at 10:00 a.m. EST in Room A315 of the Georgia World Congress Center. Gene Therapy Restores All Immune Cell Types in Infants with X-linked Severe Combined Immunodeficiency  Infants with the devastating genetic condition X-SCID… Read full this story
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