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Stem Cell Therapy: Cell‐Based Therapy for Canavan Disease Using Human iPSC‐Derived NPCs and OPCs (Adv. Sci. 23/2020)
Canavan disease (CD) is a leukodystrophy caused by aspartoacylase (ASPA) gene mutation with no cure so far. In this study, a functional ASPA was introduced into patient iPSC‐derived neural progenitor cells (iNPCs) and the engineered ASPA iNPCs could rescue major pathological features of CD in a dise...
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| 發表在: | Adv Sci (Weinh) |
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| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
John Wiley and Sons Inc.
2020
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7709969/ https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/advs.202070131 |
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