Scientists led by Dr. Craig Mello of The University of Massachusetts have developed a genetic tool – CRISPR [clustered regularly interspaced short palindromic repeats] – to revolutionize the way stem cells are applied to treat genetic diseases, such as sickle cell or thalassemia. CRISPR aims to expedite and improve upon the process of translational genomics, in which the patient’s stem cells are extracted, altered to repair the damaged gene, and then transplanted back to the patient. Continue reading
Researchers at the Cedars-Mt. Sinai Regenerative Medicine Institute, led by Dr. Robert H. Baloh, have devised a method to study and develop potential treatments for Amyotrophic Lateral Sclerosis [ALS] by utilizing patients’ stem cell-derived neurons. In an example of what is referred to as translational genomics, researchers extract autologous (the patient’s own) stem cells, correct the defective gene causing the disease, and then, in a potential treatment protocol, transplant the cells back into the patient to reverse neural degeneration. Continue reading
A team of Gladstone research scientists has utilized stem cells to develop a new gene-editing technique for inducing and studying rare genetic mutations linked to disease. The technique, called TALENs, presents an opportunity for patients with genetic disorders to use their own stem cells to identify their specific genetic mutations. Continue reading
Researchers at UMass Medical School use human stem cells to ‘shut down’ the chromosomes causing Down syndrome. The lead researcher, Jeanne B. Lawrence, a professor of cell and developmental biology at UMass Medical School, explained, “Our hope is that for individuals living with Down syndrome, this proof-of-principal opens up multiple exciting new avenues for studying the disorder now, and brings into the realm of consideration research on the concept of ‘chromosome therapy’ in the future”. The treatment seeks to address the root cause of the disease as opposed to merely mitigating the symptoms of the disease.
Gene therapies utilizing stem cells are being developed that may lead to a cure for several genetic diseases. Currently, two clinical trials were announced to treat Cerebral Adrenoleukodystrophy (CCALD) and betathalassemia/sickle cell disease.
Recently, researchers utilized stem cells to successfully treat six children with rare genetic diseases. Three of the children were born with Metachromatic Leukodystrophy (MLD), a hereditary neurodegenerative disease. The other three children were born with Wiskott-Aldrich Syndrome (WAS), a hereditary immune system disorder.