The uridine-rich 7 (U7) small nuclear RNA (snRNA) is a component of a small nuclear ribonucleoprotein (snRNP) complex. U7 snRNA naturally contains an antisense sequence that identifies histone premessenger RNAs (pre-mRNAs) and is involved in their 3′ end processing. By altering this antisense sequence, researchers have turned U7 snRNA into a versatile tool for targeting pre-mRNAs and modifying splicing. Encapsulating a modified U7 snRNA into a viral vector such as adeno-associated virus (also referred as vectorized exon skipping/inclusion, or VES/VEI) enables the delivery of this highly efficacious splicing modulator into a range of cell lines, primary cells, and tissues. In addition, and in contrast to antisense oligonucleotides, viral delivery of U7 snRNA enables long-term expression of antisense sequences in the nucleus as part of a stable snRNP complex. As a result, VES/VEI has emerged as a promising therapeutic platform for treating a large variety of human diseases caused by errors in pre-mRNA splicing or its regulation. Here we provide an overview of U7 snRNA's natural function and its applications in gene therapy.

中文翻译：

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U7 snRNA，一种对基因治疗有重大影响的小 RNA

富含尿苷的 7 (U7) 小核 RNA (snRNA) 是小核核糖核蛋白 (snRNP) 复合物的一个组成部分。U7 snRNA 天然含有一个反义序列，可识别组蛋白前信使 RNA (pre-mRNA) 并参与其 3' 末端加工。通过改变这个反义序列，研究人员已经将 U7 snRNA 变成了一种用于靶向前 mRNA 和修饰剪接的多功能工具。将修饰的 U7 snRNA 封装到病毒载体中，例如腺相关病毒（也称为矢量化外显子跳跃/包含，或 VES/VEI），可以将这种高效剪接调节剂传递到一系列细胞系、原代细胞和组织。此外，与反义寡核苷酸相比，U7 snRNA 的病毒递送使反义序列能够在细胞核中作为稳定的 snRNP 复合物的一部分长期表达。因此，VES/VEI 已成为一个有前途的治疗平台，用于治疗由前 mRNA 剪接或其调节错误引起的多种人类疾病。在这里，我们概述了 U7 snRNA 的天然功能及其在基因治疗中的应用。