Applying regenerative medicine to a common shoulder surgery could have an impact on the need for follow-up revision surgery in some patients, according to a Mayo Clinic study of real-world evidence. Mayo Clinic researchers analyzed the largest set of data available to determine if adding bone marrow aspirate concentrate to repaired tissue after standard rotator cuff surgery would improve
A potential 1-time gene editing treatment for severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) is entering approval review by the US Food and Drug Administration (FDA), the European Medicines Agency, and the UK Medicines and Healthcare products Regulatory Agency, according to a company statement. Both diseases involve variants in the gene encoding β globin. The variants
OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency Sensorion, a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, announces that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to the Company’s lead therapy gene
A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and
Cambridge researchers are taking part in the world’s first clinical trial of red blood cells that have been grown in a laboratory for transfusion into another person. The manufactured blood cells were grown from stem cells from donors. The red cells were then transfused into volunteers in the RESTORE randomised controlled clinical trial. This is
In recent years, researchers have made measurable progress, using animal models, to promote tissue regeneration in spinal cord injuries (SCI) through implanted neural stem cells or grafts. Other efforts have shown that intensive physical rehabilitation can improve function after SCI by promoting greater or new roles for undamaged or spared cells and neural circuits. In
A recent study published in the journal Nature Medicine highlights the potential of CAR T therapy beyond this realm—specifically for lupus and other autoimmune diseases. What is Lupus? Lupus (systemic lupus erythematosus) is an autoimmune disease that affects women approximately ten more than men, and is characterized by the overproduction of antibodies that attack the body’s own tissues. Lupus
Multiple myeloma is a relatively uncommon yet serious disease estimated to impact more than 30,000 US citizens this year. Although several treatment options exist, the illness is considered incurable as most treatments do not resolve the condition permanently—including the most recent advancements with CAR T cells. Here, we describe an approach using a different variant
Gene Therapy Sees Children With Rare Genetic Condition Walk And Talk For First Time Aromatic l-amino acid decarboxylase (AADC) deficiency is a genetic disorder of the nervous system. Only about 120 cases have ever been reported, and around half of these have been in people of Asian ancestry. The condition is caused by mutations in the DDC gene, which
One of the dreams of cancer therapy is to use the power of the body to heal itself. This dream, long in the making, is becoming a reality thanks to deep and fundamental understandings of the immune system, the primary means by which we protect ourselves from external and internal threats. The immune system recognizes
