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
Blood cancers like multiple myeloma have long been considered incurable, but in the last decade, the development of precision immunotherapy treatments has offered patients months or even years of extended life. One such treatment, known as CAR-T cell therapy, turns immune system T cells into killers of cancer cells. They have been so transformative that
LMU researchers demonstrate in a zebrafish model that two proteins prevent scar formation in the brain, thereby improving the ability of tissue to regenerate. Whereas cells regularly renew themselves in most endogenous tissues, the number of nerve cells in the human brain and spinal cord remains constant. Although nerve cells can regenerate in the brains
Researchers have engineered a family of adeno-associated viral vectors that cross the blood-brain barrier in primate models. Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed. Researchers have engineered viruses called adeno-associated viruses (AAVs) to deliver
Penn Medicine researchers laud early results for CAR T therapy in lupus patients, point to broader horizon for the use of personalized cellular therapies Engineered immune cells, known as CAR T cells, have shown the world what personalized immunotherapies can do to fight blood cancers. Now, investigators have reported highly promising early results for CAR
Researchers find that combining novel gene-editing CRISPR technology with CAR T therapy could simplify and improve CAR T therapy in one fell swoop. Traditional CAR T Therapy A remarkable feat in cancer care, today people with difficult-to-treat blood cancers can receive CAR T therapy, a personalized “drug” made from their own immune cells. Chimeric Antigen Receptor T cell (CAR T)
Today, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. “Gene therapy for hemophilia has
The increasing integration of CAR T-cell therapies into the treatment paradigm for adult patients with relapsed or refractory multiple myeloma addresses several unmet needs and improves outcomes for this historically limited patient population. The increasing integration of CAR T-cell therapies into the treatment paradigm for adult patients with relapsed or refractory multiple myeloma addresses several
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
