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European Hematology Association - Genetic Modification of Autologous Hematopoietic Stem Cells: A Potential Treatment for Mucopolysaccharidosis Type I Hurler

/PRNewswire/ Mucopolysaccharidosis type I Hurler (MPS-IH) is caused by a deficiency in alpha-L-iduronidase (IDUA) and is typically managed by allogeneic.

Italy
Milan
Lombardia
Netherlands
Maria-ester-bernardo
Prnewswire-mucopolysaccharidosis
European-hematology-association
Professor-maria-ester-bernardo
Professor-maria-ester
Ospedale-san-raffaele
Every-june
Annual-congress

SpliSense Presents Preclinical Data for SPL23-ASO, mRNA-Altering Drug Candidate for the Treatment of Cystic Fibrosis, at the European Cystic Fibrosis Conference

/PRNewswire/ SpliSense, a biopharmaceutical company developing transformative mRNA-altering therapies for cystic fibrosis (CF) and other pulmonary diseases,.

Jerusalem
Israel-general
Israel
Tsipi-haitovsky
Gili-hart
Hebrew-university-of-jerusalem
Cystic-fibrosis-transmembrane
Batsheva-kerem
Hebrew-university
Chief-scientific-officer
European-cystic-fibrosis-digital-conference
Splisense

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