A collaborative effort between researchers at Stanford University, the Joint Institute of Metrology and Biology, and the National Institute of Standards and Technology has developed a modified and more targeted version of CRISPR, which is more efficient at editing single nucleotide mutations. The new system is called MAGESTIC (multiplexed, accurate genome-editing through short, trackable, integrated cellular barcodes), and it has been shown to successfully modify genes by accurately targeting the location of defective genes. MAGESTIC ameliorates and addresses the current shortcomings of gene-editing technology by enhancing the ability of CRISPR to target single genes [out of millions] with the purpose of correcting specific mutations.
A genetically modified stem cell therapy for Diffuse Large B-Cell Lymphoma (DLBCL) has been approved by the FDA. Researchers at the Abramson Cancer Center, in collaboration with Novartis, have successfully administered a CAR-T Cell therapy, called Kymriah, for the most common type of non-Hodgkin Lymphoma. DLBCL is a fast growing cancer that affects B lymphocytes, which are responsible for producing antibodies that help fight infections in the body. This groundbreaking treatment involves obtaining autologous (the patient’s own) T cells, which are a more specialized type of stem cell, and genetically engineering the cells to track down and destroy cancerous cells.
A Phase I clinical trial to test the efficacy of genetically modified autologous (the patient’s own) stem cells to treat beta-thalassemia has been initiated. This condition is an inherited disease that affects the production of hemoglobin, which is responsible for carrying oxygen in the body and delivering it to tissues and vital organs. With thousands of new cases every year, this condition often results in fatigue, bone fragility and extreme anemia (a deficiency of iron in the blood). This trial aims to create a groundbreaking protocol that would obtain autologous stem cells from the patients, genetically alter them to produce the missing protein responsible for the condition and, reintroduce the stem cells back into the body through a transfusion.
A $225m investment from pharmaceutical giant Bayer catapults BlueRock Therapeutics onto the stem cell therapy stage. BlueRock will initially focus on cardiovascular and neurological treatments. A team led by Dr. Michael Laflamme and Dr. Gordon Keller will concentrate on regenerating heart muscle for patients who have suffered a heart attack, whilst a team led by Dr. Lorenz Studer and Dr. Viviane Tabar will concentrate on restoring dopamine-producing cells in patients with Parkinson's disease .
Scientists from the RIKEN Center for Developmental Biology in Japan have recently grown skin tissue from transformed stem cells. Their work demonstrated an advancement from previous efforts to grow skin in that the transplanted stem cells developed as integumentary tissue – the tissue between the outer and inner skin, which holds the functional properties of the skin, including sweat glands and hair follicles.
Scientists from the University of Nottingham and Harvard University have developed a treatment that takes advantage of the unique regenerative characteristics of stem cells to enable teeth to heal themselves. The treatment represents an advancement over the current methods to treat severe cavities by eliminating the need for root canals. By stimulating the dental stem cells [mesenchymal stem cells] within the tooth, the growth of dentin, the bony material that makes up the majority of the tooth, is encouraged. This allows patients to regrow damaged teeth effectively.
Doctors from the Melbourne Stem Cell Center are using stem cells to regrow damaged knee cartilage. Over 70 patients have had their own isolated and expanded mesenchymal stem cells (MSCs) injected into their own knee joints. In initial results, half of those treated at Melbourne Stem Cell Center saw a three-quarters reduction in pain and vastly improved knee function.
A group of researchers investigated the effectiveness of mesenchymal stem cells for several age-related neurodegenerative disorders. The team implanted the stem cells in a group of 14 patients aged between 30 and 75, including four subjects who had completely gray hair. During their investigation, they made an unusual discovery. After six weeks of stem cell implantation, the reversal of graying hair was observed for both scalp and beard hair.
A group of researchers from Newcastle University discovered a novel way to preserve and prolong lifespans of mesenchymal stem cells. Although substantial research documents the multipurpose efficacy of mesenchymal stem cell therapy in wound healing, its adoption into mainstream healthcare has been challenging. Traditionally, adipose stem cells extracted from patients have to be handled in specialized conditions as they do not survive well outside the body. The Newcastle researchers encapsulated the stem cells in an alginate gel, prolonging the cells’ lives for up to three days in ambient temperatures and conditions. This protocol may offer a simple solution to the quagmire of transporting adipose cell cultures for treatment options.
Researchers at the University of Miami Miller School of Medicine have found that age has a profound effect on the efficacy of transplanted mesenchymal stem cell therapy. A series of previous studies had shown that stem cells could repair damage done to lungs caused by pulmonary fibrosis, but those studies relied on donor cells from relatively young animals. This study examined the differences in young and old stem cells, finding substantial differences in their biological utility.