ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Mucopolysaccharidosis type I (MPS I) is a severe disease due to deficiency of the lysosomal hydrolase α-L-iduronidase (IDUA) and the subsequent accumulation of the glycosaminoglycans (GAG), leading to progressive, systemic disease and a shortened lifespan. Current treatment options consist of hemato...

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Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Mol Ther
Päätekijät: Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michael J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, Whitley, Chester B.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2019
Aiheet:
Original Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC6319315/
https://ncbi.nlm.nih.gov/pubmed/30528089
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.10.018
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