Regenerative Medicine and Stem Cells Technologies

The process of making functional brain cells in a lab dish requires the precise activation of selfish genetic elements known as LINE-1 (L1) retrotransposons. The finding, from researchers at KAUST, could lead to safer and more effective regenerative therapies for Parkinson's disease and other brain conditions.

In the U.S., someone has a heart attack every 40 seconds, and yet researchers have not had a model that fully mimics what occurs in the human heart after a heart attack.

Researchers at Penn Medicine (PA, USA) have uncovered new roles of autophagy in embryonic stem cell renewal and differentiation. These processes, which pertain to chaperone-mediated autophagy (CMA) and a related metabolite, offer new insights that may be critical in the development of novel regenerative therapies. The study was recently published in Science.

FDA today approved the third chimeric antigen receptor (CAR) T-cell therapy, this time granting a green light for Kite Pharma’s brexucabtagene autoleucel, the first cell-based gene therapy to treat relapsed or refractory mantle cell lymphoma (MCL).

In the U.S., someone has a heart attack every 40 seconds, and yet researchers have not had a model that fully mimics what occurs in the human heart after a heart attack.

Skeletal muscle can be functionally compromised by genetic myopathies, aging, traumatic injuries and tumor ablation. Under some conditions, such as severe traumatic injuries and volumetric muscle loss, the regeneration process is significantly hindered by fibrous scar tissue formation and therefore causing muscle dysfunction.

A massive research effort over more than a quarter century has tried to make personalized blood stem cells for use in treating leukemias, among many other uses.

In regenerative medicine, an ideal treatment for patients whose muscles are damaged from lack of oxygen would be to invigorate them with an injection of their own stem cells.

Switching what the powerhouses of heart cells consume for energy could help the heart regenerate when cells die, a new study led by UT Southwestern researchers suggests.

Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company has received both orphan drug designation and rare pediatric disease designation from the U.S Food and Drug Administration (FDA) for OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I).