Shchaslyvyi Aladdin Y, Antonenko Svitlana V, Tesliuk Maksym G, Telegeev Gennadiy D
Institute of Molecular Biology and Genetics, National Academy of Sciences of Ukraine, 150, Zabolotnogo Str., 03143 Kyiv, Ukraine.
Pharmaceuticals (Basel). 2023 Oct 5;16(10):1416. doi: 10.3390/ph16101416.
In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments, are now being addressed at their root cause-the genetic level. Presently, the gene therapy landscape stands adorned with 22 approved in vivo and ex vivo products, including IMLYGIC, LUXTURNA, Zolgensma, Spinraza, Patisiran, and many more. In this comprehensive exploration, we delve into a rich assortment of 16 drugs, from siRNA, miRNA, and CRISPR/Cas9 to DNA aptamers and TRAIL/APO2L, as well as 46 carriers, from AAV, AdV, LNPs, and exosomes to naked mRNA, sonoporation, and magnetofection. The article also discusses the advantages and disadvantages of each product and vector type, as well as the current challenges faced in the practical use of gene therapy and its future potential.
在基因治疗领域，1972年保罗·伯格首次成功鉴定出重组DNA，这一具有开创性的事件成为了一个关键时刻。这一成就为未来的突破奠定了基础。曾经被认为无法战胜的疾病，如黑色素瘤、胰腺癌和许多其他疾病，现在正从根源——基因层面进行攻克。目前，基因治疗领域已有22种体内和体外批准产品，包括IMLYGIC、LUXTURNA、Zolgensma、Spinraza、Patisiran等等。在本次全面探讨中，我们深入研究了16种丰富多样的药物，从小干扰RNA、微小RNA和CRISPR/Cas9到DNA适配体以及肿瘤坏死因子相关凋亡诱导配体/APO2L，还研究了46种载体，从腺相关病毒、腺病毒、脂质纳米颗粒和外泌体到裸mRNA、声穿孔和磁转染。本文还讨论了每种产品和载体类型的优缺点，以及基因治疗实际应用中目前面临的挑战及其未来潜力。
