Engineered CRISPR Systems for Next Generation Gene Therapies
作者： Michael Pineda , Farzaneh Moghadam , Mo R. Ebrahimkhani , Samira Kiani
DOI: 10.1021/ACSSYNBIO.7B00011
关键词:
摘要: An ideal in vivo gene therapy platform provides safe, reprogrammable, and precise strategies which modulate cell tissue regulatory networks with a high temporal spatial resolution. Clustered regularly interspaced short palindromic repeats (CRISPR), bacterial adoptive immune system, its CRISPR-associated protein 9 (Cas9), have gained attention for the ability to target modify DNA sequences on demand unprecedented flexibility precision. The precision programmability of Cas9 is derived from complexation guide-RNA (gRNA) that complementary desired genomic sequence. CRISPR systems open-up widespread applications including genetic disease modeling, functional screens, synthetic regulation. plausibility engineering using has garnered significant traction as next generation therapeutic. However, there are hurdles need be addressed before CRISPR-based fully implemented. Some key issues cen...
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