Long-term hematopoietic stem cell lentiviral gene therapy rescues neuromuscular manifestations in preclinical study of Pompe disease mice
| Autor: | van Til, Niek P., Schindler, Jeffrey W., Yoon, John K., Dogan, Yildirim, Barese, Cecilia, Unnisa, Zeenath, Guda, Swaroopa, Jacobs, Mary E., Pizzurro, Daniella, Trame, Mirjam, Maiwald, Tim, Oborski, Christine, Rothe, Michael, Schambach, Axel, Harper, Claudia, Pfeifer, Richard, Mason, Chris |
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| Zdroj: | In Molecular Genetics and Metabolism February 2022 135(2):S122-S123 |
| Databáze: | ScienceDirect |
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