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Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa
Targeted transcriptional modulation in the central nervous system (CNS) can be achieved by adeno-associated virus (AAV) delivery of CRISPR activation (CRISPRa) and interference (CRISPRi) transgenes. To enable AAV packaging, we constructed minimal CRISPRa and CRISPRi transgenes by fusing catalyticall...
Autores principales: | Lau, Cia-Hin, Ho, Jonathan Weng-Thim, Lo, Pik Kwan, Tin, Chung |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6526230/ https://www.ncbi.nlm.nih.gov/pubmed/31108320 http://dx.doi.org/10.1016/j.omtn.2019.04.015 |
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