According to Medical Xpress, gene therapy may become the next treatment option for those with chronic kidney disease, CKD. Researchers at Washington University School of Medicine in St. Louis have effectively shown in mice that genetic material is able to be sent to damaged kidney cells in order to repair them. As a result, this gene therapy could be soon applied to humans with CKD as well.
CKD Explained
Chronic kidney disease, abbreviated as CKD, is lasting damage to the kidneys that gets worse and worse over time. The kidneys are the body’s organs that are responsible for filtering wastes and excess fluids from the blood. In those with chronic kidney disease, there is often an unhealthy amount of buildup of these wastes and fluids that eventually cause health complications. Symptoms of CKD include jaundice, fatigue and weakness, sleep issues, persistent itching, shortness of breath, and high blood pressure among others. There are a few treatment options for those with CKD that involve mainly controlling symptoms. To read more about these and learn more about chronic kidney disease, click here.
What Researchers Think
Researchers involved in this science believe that using gene therapy could effectively send genes that potentially reverse the cell damage that usually leads to chronic kidney disease.
“Chronic kidney disease is an enormous and growing problem,” said author Benjamin D. Humphreys. “Unfortunately, over the years, we haven’t developed more effective drugs for the condition, and this reality is leading us to explore gene therapy.”
More About CKD
Other conditions are capable of causing chronic kidney failure, including diabetes and hypertension. As a result of this and the other symptoms of these conditions, many of those affected by CKD don’t even realize they have it until they experience irreparable organ damage.
Even more seriously, advanced and aggressive CKD can lead to cardiovascular disease. Those patients who experience kidney failure may also be at higher risk for complications from cardiovascular disease. This is a scary reality, but researchers are hopeful that positive changes are on the horizon.
“Part of the reason there have been so few advances in kidney disease treatment is because the kidney is complex, and we don’t fully understand the disease process,” said Humphreys. “However, scientists are making progress, and I am optimistic.”
The Research
To delve right into things, Humphreys and his team, in collaboration with teams from Harvard and MIT, tried to figure out if adeno-associated virus (AAV) was capable of delivering important genetic material to specific kidney cells. This type of genetic material delivery has never been studied before, so this research is the first experimental data on this approach.
Scientists focused on six different AAV viruses in both stem-cell-derived human kidney organoids and mice. One type of the virus, Anc80, proved to be successful in delivering material to two different cells that play a role in CKD. The fact that the correct genetic material was appropriately transferred to the target kidney cells in the study was a huge step forward for researchers.
“This was a happy surprise,” said Humphreys. “We were not expecting this.”
Not Too Soon
All this being said, the researchers cautioned that the data and research is still early on in its development. In the future, researchers realize they will need to find a gene capable of extensively correcting kidney cells that are damaged. The future for this research, however, still seems very exciting.
“The interesting thing about the adeno-associated viruses is that they persist in the body for many months, potentially giving a therapeutic gene a chance to do its work,” Humphreys explained. “Chronic kidney disease is a slowly progressive disease so that is an advantage. After many more years of research, we could envision that patients would need injections maybe twice a year as opposed to every week, like with chemo.”
Since there is such limited information involving kidney diseases and treatment, this research is clearly a big step forward in the right direction.
