U7 snRNA, a Small RNA with a Big Impact in Gene Therapy
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 s...
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| Veröffentlicht in: | Human gene therapy 2021-11, Vol.32 (21-22), p.1317-1329 |
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| container_title | Human gene therapy |
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| creator | Lesman, Daniel Rodriguez, Yacidzohara Rajakumar, Dhanarajan Wein, Nicolas |
| description | 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. |
| doi_str_mv | 10.1089/hum.2021.047 |
| format | Article |
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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. 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| source | MEDLINE; Alma/SFX Local Collection |
| subjects | Antisense oligonucleotides Cell lines Exon skipping Gene therapy Genetic Therapy Genetic Vectors Histones Humans mRNA Oligonucleotides Ribonucleic acid Ribonucleoproteins (small nuclear) RNA RNA Precursors RNA, Small Nuclear - genetics snRNA Splicing Uridine Viruses |
| title | U7 snRNA, a Small RNA with a Big Impact in Gene Therapy |
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