Blocking an immune-regulating protein reverses the damage caused by acute and chronic kidney disease, a preclinical study suggests. Led by scientists at Duke-NUS Medical School and the National Heart Centre Singapore, researchers in Singapore and Germany have found that renal tubular cells, which line the tiny tubes inside kidneys, release a scar-regulating protein called interleukin-11
In recent months there have been some major jumps—unprecedented success stories—that indicate our ability to engineer T cells may well have a substantial impact for multiple medical conditions that have not been responsive to conventional therapies or for which there is no available treatment. This can be regarded as the quintessential individualized medicine intervention—specifically modifying
Can gene therapy trials for vision loss, hemophilia, and melanoma break through in 2023? fter another turbulent year in gene therapy development, all eyes are on five major trial readouts set for H1 2023. The clinical studies target rare blood disorders, inherited retinal diseases, and metastatic melanoma—each of which poses distinct trial design challenges. First, two small
An innovative immunotherapy combination has shown a stunning 99% response rate in children with relapsed leukemia. The phase 2 trial, run jointly between researchers at St. Jude Children’s Research Hospital in Memphis and Shanghai Children’s Hospital in China tested the therapy in 225 children who had relapsed after conventional treatment. The work published in the Journal
New immunotherapies can allow healing cells to be activated or deactivated according to the stage of the disease, while more accurately targeting tumors While CAR T therapy represents one of the most impressive innovations in cancer care, the treatment can cause autoimmune-like side effects. Many have contended with the dilemma of maximizing the therapy’s benefits
Pfizer announced positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion
Implantable platform provides prolonged treatment of Type 1 diabetes A quarter-sized device created at Houston Methodist could drastically alter the course of treatment for Type 1 diabetes, a chronic condition that impacts millions of Americans and does not have a cure. In a study published in the Dec. 26 issue of Nature Communications, a research team led
H.T. Begay is a happy kid. He’s smiley, silly, and definitely trying to make you laugh. The four-year-old’s two neat braids of dark hair wave behind him as he runs among the dust, dogs, and sheep near his family’s ranch on their Navajo reservation in Arizona. On a table inside the family’s sweat lodge is
CAR T therapy can treat blood cancers by inserting new genes into a patient’s own immune cells using viruses. Early clinical trial results present an alternative that forgoes viral gene transfer: CRISPR technology. Such integration of CRISPR gene editing could improve the precision, speed and cost-effectiveness of CAR T cell production. In addition, researchers hope
A newly discovered link between protein misfolding and liver cancer could help improve gene therapy for hemophilia Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world’s deadliest diseases. The findings, published
