Simple genetic test by TGen reveals likely causes of disease, after other extensive testing failed; 1 child's case produces discovery PHOENIX, Ariz. -- April 9, 2015 -- Scientists at the Translational Genomics Research Institute (TGen), using state-of-the-art genetic technology, have discovered the likely cause of a child's rare type of severe muscle weakness. The child was one of six cases in which TGen sequenced -- or decoded -- the genes of patients with Neuromuscular Disease (NMD) and was then able to identify the genetic source, or likely genetic source, of each child's symptoms, according to a study published April 8 in the journal Molecular Genetics & Genomic Medicine. "In all six cases of myopathy, or muscle weakness, these children had undergone extensive, expensive and invasive testing -- often over many years -- without a successful diagnosis, until they enrolled in our study," said Dr. Lisa Baumbach-Reardon, an Associate Professor of TGen's Integrated Cancer Genomics Division and the study's senior author. This is a prime example of the type of "personalized medicine" TGen uses to zero in on diagnoses for patients, and to help their physicians find the best possible treatments. "Our results demonstrate the diagnostic value of a comprehensive approach … Continue reading →

Scientists at the Translational Genomics Research Institute (TGen), using state-of-the-art genetic technology, have discovered the likely cause of a child's rare type of severe muscle weakness. The child was one of six cases in which TGen sequenced -- or decoded -- the genes of patients with Neuromuscular Disease (NMD) and was then able to identify the genetic source, or likely genetic source, of each child's symptoms, according to a study published April 8 in the journal Molecular Genetics & Genomic Medicine. "In all six cases of myopathy, or muscle weakness, these children had undergone extensive, expensive and invasive testing -- often over many years -- without a successful diagnosis, until they enrolled in our study," said Dr. Lisa Baumbach-Reardon, an Associate Professor of TGen's Integrated Cancer Genomics Division and the study's senior author. This is a prime example of the type of "personalized medicine" TGen uses to zero in on diagnoses for patients, and to help their physicians find the best possible treatments. "Our results demonstrate the diagnostic value of a comprehensive approach to genetic sequencing," said Dr. Baumbach-Reardon. "This type of next-generation sequencing can greatly improve the ability to identify pathogenic, or disease-causing, genetic variants with a single, timely, … Continue reading →

New Delhi, April 11 (IANS): Though there are many stem cell therapies that are being explored, bone marrow transplant is the only one which has been tried and tested, a noted scientist has said. Stem cell therapy is the use of stem cells to treat or prevent a disease or condition. Stem cells are undifferentiated cells of an organism which are capable of giving rise to indefinitely more cells of the same type, and from which certain other kinds of cells arise by differentiation. "Most of the stem cell therapies like the ones which can successfully treat Parkinson's disease and methods of ortho-reconstruction are in experimental stages," noted cellular biologist Jyotsna Dhawan said in a special lecture on 'Stem cells: Myths and Realities' at the India International Centre (IIC) here Friday evening. Dhawan, a senior scientist at the Centre for Cellular and Molecular Biology, Hyderabad, said stem cells are a potential source of repair of other tissues and can be very useful in treating genetic and inherited diseases. She said stem cells are specially useful in treating genetic disorders associated with blood like haemophilia and thalassemia. "But, major ethical issues arise when embryonic stem cells (cells taken from a foetus) … Continue reading →

(MENAFN - ProactiveInvestors) Regeneus (ASX: RGS) has achieved a key milestone with the manufacture of its off-the-shelf stem cell therapy product Progenza for its First-in-Human trial for the treatment of osteoarthritis. The company is on track to receive ethics approval and commence recruitment for the trial in the first-half of 2015. Adding interest the company highlighted the benefit of using adipose or fat tissue over other tissue types by demonstrating the capacity to produce millions of therapeutic doses of Progenza from a single donor. The production of commercial quantities of stem cells from a single donor is critical to maximise dose-to-dose consistency chief executive officer John Martin said. This scale of production will minimise clinical trial and regulatory risks while reducing the cost of the final product. One of the key advantages for manufacturing Progenza at industrial scale is that it uses stem cells sourced from adipose or fat tissue. Adipose tissue is readily available from donors in large quantities and has significantly higher stem cells per gram of tissue than other tissue sources such as bone marrow or cord tissue. Also adipose derived stem cells show greater capacity for expansion than stem cells from other tissue types. Progenza adipose … Continue reading →