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The Nature subjournal identified gene therapy targets for polycystic kidney disease

 Last Update: 2022-10-03
 Source: Internet
 Author: User

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Image: This image shows a severe cystic kidney from a mouse model of ADPKD (left

Researchers at the University of Texas at Southwestern report that blocking inhibition of PKD1 and PKD2 gene expression by deleting binding sites of microRNAs can hinder the formation and growth

"For 25 years, we have known that ADPKD is caused

ADPKD is one of the most common human genetic diseases and the most common genetic cause of kidney failure, affecting approximately 12.

Proteins are produced (or translated) by the gene's messenger,

PKD1 contains the binding site of miR-17, a microRNA

The researchers removed the miR-17 binding site

"In many genetic conditions, one copy of the gene is mutated, but the other copy is still normal

The University of Texas Southwestern opened the PKD and Genetic Nephropathy Clinic in 2016, co-led

Other researchers who contributed to the study include Patricia copo - stark, Laurence Biggers, Andrea Flaten and Jesus Alvarez, all from UTSW; Chunmin Zhang, Tania Valencia, Darren P.

This work was supported by grants

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