National Key Laboratory of Crop Genetic Improvement, Huazhong Agricultural University, Wuhan, P.R., China.
Women Medical and Dental College, Khyber Medical University, Khyber Pakhtunkhwa, Pakistan.
Curr Probl Cardiol. 2023 Jul;48(7):101692. doi: 10.1016/j.cpcardiol.2023.101692. Epub 2023 Mar 9.
Over the past 2 decades, genome-editing technique has proven to be a robust editing method that revolutionizes the field of biomedicine. At the genetic level, it can be efficiently utilized to generate various disease-resistance models to elucidate the mechanism of human diseases. It also develops an outstanding tool and enables the generation of genetically modified organisms for the treatment and prevention of various diseases. The versatile and novel clustered regularly interspaced short palindromic repeats (CRISPR/Cas9) system mitigates the challenges of various genome editing techniques such as zinc-finger nucleases, and transcription activator-like effector nucleases. For this reason, it has become a ground-breaking technology potentially employed to manipulate the desired gene of interest. Interestingly, this system has been broadly utilized due to its tremendous applications for treating and preventing tumors and various rare disorders; however, its applications for treating cardiovascular diseases (CVDs) remain in infancy. More recently, 2 newly developed genome editing techniques, such as base editing and prime editing, have further broadened the accuracy range to treat CVDs under consideration. Furthermore, recently emerged CRISPR tools have been potentially applied in vivo and in vitro to treat CVDs. To the best of our knowledge, we strongly enlightened the applications of the CRISPR/Cas9 system that opened a new window in the field of cardiovascular research and, in detail, discussed the challenges and limitations of CVDs.
在过去的 20 年中，基因组编辑技术已被证明是一种强大的编辑方法，彻底改变了生物医学领域。在基因水平上，它可以有效地用于生成各种抗病模型，以阐明人类疾病的发病机制。它还开发了一种出色的工具，使基因修饰生物体的产生用于治疗和预防各种疾病成为可能。多功能且新颖的成簇规律间隔短回文重复序列 (CRISPR/Cas9) 系统减轻了各种基因组编辑技术（如锌指核酸酶和转录激活因子样效应物核酸酶）的挑战。出于这个原因，它已成为一种革命性的技术，可用于操纵所需的感兴趣基因。有趣的是，由于其在治疗和预防肿瘤和各种罕见疾病方面的巨大应用，该系统已被广泛应用；然而，其在治疗心血管疾病 (CVD) 方面的应用仍处于起步阶段。最近，两种新开发的基因组编辑技术，如碱基编辑和 Prime 编辑，进一步拓宽了考虑治疗 CVD 的准确性范围。此外，最近出现的 CRISPR 工具已被潜在地应用于体内和体外以治疗 CVD。据我们所知，我们强烈启发了 CRISPR/Cas9 系统的应用，为心血管研究领域开辟了新的窗口，并详细讨论了 CVD 的挑战和局限性。
