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|a UAMI
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|a Severino, Valeria.
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|a Tools and Applications in Gene Therapy
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|a Ashland :
|b Delve Publishing,
|c 2019.
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| 300 |
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|a 1 online resource (366 pages)
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|a Cover; Half Title Page; Title Page; Copyright Page; Declaration; About the Editor; Table of Contents; List of Contributors; List of Abbreviations; Preface; Chapter 1 Gene Therapy: Progress and Predictions; Abstract; Introduction; Gene Therapy Remains A Delivery Challenge; Replacement Gene Therapy Using Bone Marrow Transplantation; Direct Injection of Adeno-Associated Virus Vectors For Gene Replacement Therapy; Gene Therapy to Treat Cancer And Infectious Disease; Gene Therapy as a Different Formulation of a Conventional Medicine; Where Will Gene Therapy Be In Another 25 Years?; References
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|a Chapter 2 Advances In Non-Viral DNA Vectors For Gene TherapyAbstract; Introduction to Gene Therapy; Challenges of Using Plasmid Vectors For Gene Therapy; Improvements to Plasmid Vectors; Development of Minicircles and Minivectors; Therapeutic Needs Best Addressed by Minimized Vectors; Concluding Remarks: Moving Minimized Non-Viral DNA Vectors Into The Clinic; Acknowledgments; Author Contributions; References; Chapter 3 Gene Delivery Technologies For Efficient Genome Editing: Applications In Gene Therapy; Abstract; Introduction; Viral-Based Vectors; Non-Viral-Based Vectors; Acknowledgements
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| 504 |
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|a ReferencesChapter 4 Alpharetroviral Vectors: From A Cancer-Causing Agent To A Useful Tool For Human Gene Therapy; Abstract; Introduction; History of (Alpha-) Retroviruses; From The Virus To The Vector; On The Road To Clinical Applicability: Production Perspectives of Alpharetroviral Vectors; From Bench To Bedside: Regulatory Requirements For Clinical Translation; Potential Future Clinical Applications of Alpharetroviral Sin Vectors; Conclusions And Outlook; Acknowledgments; References And Notes
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| 505 |
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|a Chapter 5 The Use of The Humanized Mouse Model In Gene Therapy And Immunotherapy For HIV and CancerAbstract; Introduction; Peripheral Cell-Based Immunotherapy Modeling In Humanized Mice; Stem Cell-Based Gene Therapy In Humanized Mice; PD-1 and IFN-I Blockade Therapy For HIV and Cancer; Future Directions; Author Contributions; References; Chapter 6 Innovative Therapeutic Strategies For Cystic Fibrosis: Moving Forward To CRISPR Technique; Abstract; Introduction; Genetics; Therapy; Repairing The CFTR Mutation: Gene Correction With Genome-Editing Nucleases; Conclusion; Author Contributions
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| 504 |
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|a ReferencesChapter 7 Targeted Genome Engineering In Human Induced Pluripotent Stem Cells From Patients With Hemophilia B Using The CRISPR-Cas9 System; Abstract; Background; Methods; Results; Discussion; Conclusions; References; Chapter 8 Production of Wilson Disease Model Rabbits With Homology-Directed Precision Point Mutations In The ATP7B Gene Using The CRISPR/Cas9 System; Abstract; Introduction; Methods; Results; Discussion; Acknowledgements; References; Chapter 9 CRISPR-Cas9 HDR System Enhances AQP1 Gene Expression; Abstract; Introduction; esults; Discussion; Materials And Methods
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| 504 |
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|a References
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| 590 |
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|a eBooks on EBSCOhost
|b EBSCO eBook Subscription Academic Collection - Worldwide
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| 650 |
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0 |
|a Gene therapy.
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| 650 |
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|a Genetic Therapy
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| 650 |
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6 |
|a Thérapie génique.
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|a Gene therapy.
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|a Severino, Valeria.
|t Tools and Applications in Gene Therapy.
|d Ashland : Delve Publishing, ©2019
|
| 856 |
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