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CRISPR/Cas9-Mediated in vivo Genetic Correction in a Mouse Model of Hemophilia A
Hemophilia A (HA), a common bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII), has long been considered an attractive target for gene therapy studies. However, full-length F8 cDNA cannot be packaged efficiently by adeno-associated virus (AAV) vectors. As the second most pre...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8415270/ https://www.ncbi.nlm.nih.gov/pubmed/34485274 http://dx.doi.org/10.3389/fcell.2021.672564 |