Open access peer-reviewed Edited Volume
Site-specific endonucleases create double-strand breaks within the genome and can be targeted to literally any genetic mutation. Together with a repair template, a correction of the defective locus becomes possible. This book offers insight into the modern tools of genome editing, their hurdles and their huge potential. A new era of in vivo genetic engineering has begun.
Order hardcopy7769 Total Chapter Downloads
2 Crossref Citations
3 Dimensions Citations
chapters | Downloads | |
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Open access peer-reviewed 1. Next-Generation Therapeutics: mRNA as a Novel Therapeutic Option for Single-Gene DisordersBy Tatjana Michel, Hans-Peter Wendel and Stefanie Krajewski | 1525 | |
Open access peer-reviewed 2. Gene Delivery Technologies for Efficient Genome Editing: Applications in Gene TherapyBy Francisco Martin, Sabina Sánchez-Hernández, Alejandra Gutierrez- Guerrero and Karim Benabdellah | 988 | |
Open access peer-reviewed 3. DNA Elements Tetris: A Strategy for Gene CorrectionBy Colette Bastie and Florence Rouleux-Bonnin | 603 | |
Open access peer-reviewed 4. Gene Correction Technology and Its Impact on Viral Research and TherapyBy Guan-Huei Lee and Myo Myint Aung | 666 | |
Open access peer-reviewed 5. Gene Editing in Adult Hematopoietic Stem CellsBy Sergio López-Manzaneda, Sara Fañanas-Baquero, Virginia Nieto- Romero, Francisco-Jose Roman-Rodríguez, Maria Fernandez-Garcia, Maria J. Pino-Barrio, Fatima Rodriguez-Fornes, Begoña Diez- Cabezas, Maria Garcia-Bravo, Susana Navarro, Oscar Quintana- Bustamante and Jose C. Segovia | 1028 | |
Open access peer-reviewed 6. Insulin Gene Therapy for Type 1 Diabetes Mellitus: Unique Challenges Require Innovative SolutionsBy Andrew M Handorf, Hans W Sollinger and Tausif Alam | 1090 | |
Open access peer-reviewed 7. Application of Genome Editing Technology to MicroRNA Research in MammaliansBy Lei Yu, Jennifer Batara and Biao Lu | 1101 | |
Open access peer-reviewed 8. Emerging Gene Correction Strategies for Muscular Dystrophies: Scientific Progress and Regulatory ImpactBy Houria Bachtarzi and Tim Farries | 768 |
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