A recent clinical trial conducted at Great Ormond Street Hospital in London, England, has successfully treated seven patients suffering from the genetic disorder Wiskott-Aldrich Syndrome by utilizing autologous [the patient’s own] stem cells. The therapy was an example of translational genomics, in which doctors extract autologous stem cells, correct the faulty gene that causes Wiskott-Aldrich Syndrome, and then implant the stem cells back into the patients to produce new, healthy cells.
StemSave Blog
Wiskott-Aldrich Syndrome Cured by Stem Cell Therapy
Posted by taylor@stemsave.com on May 22, 2015 9:37:34 AM
Topics: Wiskott-Aldrich Syndrome, bank your stem cells, translational genomics, stem cell therapy, Genetic Diseases, research, StemSave, regenerative medicine, stem cells
Today, stem cells are rightfully perceived as the future of regenerative medicine, set to bring the marvels of science fiction into reality. But in looking ahead at all of the promise that stem cells hold for the future, it becomes easy to miss the scientific advances made to date for the millions of people around the world suffering from disease, trauma, and injury. Thus, today marks Stem Cell Awareness Day: a global celebration of stem cell research coordinated to highlight the treatments and therapies currently in development to create personalized regenerative therapies for patients.
Topics: neural stem cells, clinical trials, Stemcells, translational genomics, autologousstemcells, dentalstemcells, dental pulp stem cells, nanotechnology, young stem cells, stemsaveblog
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.
Topics: ALS, Phase III, neural stem cells, clinical trials, Debilitating Diseases, Stemcells, translational genomics, autologousstemcells, stemsaveblog
Genetic Diseases Decoded Utilizing Stem Cells
Posted by pamela@stemsave.com on Apr 2, 2014 6:53:06 AM
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.
Topics: clinical trials, Debilitating Diseases, Stemcells, translational genomics, autologousstemcells, stemsaveblog
Advances in Stem Cell Therapy for Down Syndrome
Posted by anna@stemsave.com on Aug 12, 2013 9:11:35 AM
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.
Topics: Stemcells, translational genomics, Down syndrome, stemsaveblog
Stem Cell Clinical Trials to Treat Genetic Disorders Announced
Posted by pamela@stemsave.com on Aug 1, 2013 8:03:20 AM
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 beta-thalassemia/sickle cell disease.
Topics: sickle cell disease, Phase III, Cerebral Adrenoleukodystrophy, clinical trials, Beta-thalassemia, Stemcells, translational genomics, autologousstemcells, young stem cells, stemsaveblog
Rare Genetic Diseases Cured with Stem Cells
Posted by pamela@stemsave.com on Jul 15, 2013 10:29:17 AM
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.
Topics: Wiskott-Aldrich Syndrome, Metachromatic Leukodystrophy, Stemcells, translational genomics, autologousstemcells, stemsaveblog