New Therapy in Development for PKD

According to a story from news-medical.net, a team of researchers have developed a potential new treatment for polycystic kidney disease (PKD), a rare disorder that ultimately causes organ failure. The drug, which is currently known as anti-miR-17 oligonucleotide RGLS4326, has undergone testing in animal models with promising results. The results of these tests were first published in the scientific journal Nature Communications.

About Polycystic Kidney Disease

Polycystic kidney disease (PKD) is the most widespread monogenic human disorder that can be potentially fatal. However, it is still generally rare. It is characterized by the development of cysts in the kidneys and sometimes other organs as well. The disease comes in an autosomal dominant form, which is the most common, and an autosomal recessive form as well. The autosomal dominant form PKD is caused by mutations affecting the PKD1 gene in 85 percent of cases; in the remainder, the mutation affects the PKD2 gene. The recessive variant is linked to the PKHD1 gene. The presentation of the disease varies considerably, even within families. Signs of kidney problems often do not appear until middle age, although cysts may begin formation very early in life. Symptoms include anemia, bloody urine, acute loin pain, uremia, liver cysts, berry aneurysm, and high blood pressure. Treatment options are varied; aquaretics can slow disease progression temporarily. Other options include a number of surgical procedures, dialysis, and, when the kidneys begin to fail, kidney transplant. To learn more about PKD, click here.

Promising Findings

When the drug was tested in a mouse model of PKD, it was capable of bringing down the size of the kidneys by as much as 50 percent. The creation of the new, experimental drug was based on the most up to date knowledge about the mechanisms of the disease. Prior studies have shown that the RNA fragment microRNA-17 is found at elevated levels in patients with PKD. This abnormality interferes in the beneficial functions of other pieces of RNA, which ultimately leads to the appearance of the characteristic cysts. The new drug is designed to inhibit the expression of microRNA-17.

The development team included scientists from Regulus Therapeutics Inc. and UT Southwestern. Check out the original study here.


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