التفاصيل البيبلوغرافية
| العنوان: |
In Vivo Genome Editing as a Therapeutic Approach |
| المؤلفون: |
Beatrice Xuan Ho, Sharon Jia Hui Loh, Woon Khiong Chan, Boon Seng Soh |
| المصدر: |
International Journal of Molecular Sciences; Volume 19; Issue 9; Pages: 2721 |
| بيانات النشر: |
Multidisciplinary Digital Publishing Institute |
| سنة النشر: |
2018 |
| المجموعة: |
MDPI Open Access Publishing |
| مصطلحات موضوعية: |
in vivo, genome editing, correcting genetic mutations, ZFN, TALENs, Cas9 |
| جغرافية الموضوع: |
agris |
| الوصف: |
Genome editing has been well established as a genome engineering tool that enables researchers to establish causal linkages between genetic mutation and biological phenotypes, providing further understanding of the genetic manifestation of many debilitating diseases. More recently, the paradigm of genome editing technologies has evolved to include the correction of mutations that cause diseases via the use of nucleases such as zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALENs), and more recently, Cas9 nuclease. With the aim of reversing disease phenotypes, which arise from somatic gene mutations, current research focuses on the clinical translatability of correcting human genetic diseases in vivo, to provide long-term therapeutic benefits and potentially circumvent the limitations of in vivo cell replacement therapy. In this review, in addition to providing an overview of the various genome editing techniques available, we have also summarized several in vivo genome engineering strategies that have successfully demonstrated disease correction via in vivo genome editing. The various benefits and challenges faced in applying in vivo genome editing in humans will also be discussed. |
| نوع الوثيقة: |
text |
| وصف الملف: |
application/pdf |
| اللغة: |
English |
| العلاقة: |
Molecular Biology; https://dx.doi.org/10.3390/ijms19092721Test |
| DOI: |
10.3390/ijms19092721 |
| الإتاحة: |
https://doi.org/10.3390/ijms19092721Test |
| حقوق: |
https://creativecommons.org/licenses/by/4.0Test/ |
| رقم الانضمام: |
edsbas.DB22D947 |
| قاعدة البيانات: |
BASE |
