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Six patients with rare blood disease are doing well after gene therapy clinical trial

Researchers have reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan — had to rely on bone marrow donations for a chance at remission.

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New protocol to generate intestinal organoids in vitro

Researchers have developed a new way to generate groups of intestinal cells that can be used, among others, to make disease models in the lab to test treatments for diseases affecting the gastrointestinal system. Using human induced pluripotent stem cells, this novel approach combined a variety of techniques that enabled the development of three-dimensional groups of intestinal cells called organoids in vitro, which can expand disease treatment testing in the lab using human cells.

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Stem cell therapy helps broken hearts heal in unexpected way

A study shows stem cell therapy helps hearts recover from a heart attack, although not for the biological reasons originally proposed two decades ago that today are the basis of ongoing clinical trials. The study reports that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.

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Tendon stem cells could revolutionize injury recovery

The buildup of scar tissue makes recovery from torn rotator cuffs, jumper’s knee, and other tendon injuries a painful, challenging process, often leading to secondary tendon ruptures. New research reveals the existence of tendon stem cells that could potentially be harnessed to improve tendon healing and even to avoid surgery.

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New method to purify cell types to high purity

Current biology research relies on the ability to purify cell types using antibodies or transgenic constructs. However, antibody availability is often limited, and genetic manipulation is labor intensive or sometimes impossible. To date, no universal method exists to enrich for cell types without a priori knowledge of cell type markers. Here, we propose GateID, a computational method that combines single-cell transcriptomics with FACS index sorting to purify cell types using only native cellular properties.

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Potential liver cancer treatment by targeting cancer stem-like cells

Liver cancer is the second leading cause of cancer-related deaths worldwide. The most common primary liver cancer in adults is known as hepatocellular carcinoma (HCC), and accounts for approximately 780,000 deaths every year. Even with advanced surgical treatments or transplantation, the 5-year survival rate for HCC patients remains poor due to frequent recurrence.

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Transient wave of hematopoietic stem cell production in late fetuses and young adults

A major challenge in regenerative medicine is producing tailor-made hematopoietic stem cells (HSCs) for transplantation. For this we need a better understanding of where, when and how HSCs are produced in vivo. Scientists have now discovered a new hematopoietic wave in the bone marrow that fills the gap between embryonic blood production and adult bone marrow hematopoietic production.

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