Safety, innovation and affordability need not be mutually exclusive goals for cutting-edge treatments. Source NYT Spinal muscular atrophy is a condition often fatal by age 2. Babies with the disorder have a rare genetic mutation that prevents the nervous system from controlling certain muscles. As they grow to be toddlers, these children struggle to move their limbs,
Source STAT Seizing on the surging popularity of at-home DNA testing kits, top academic medical institutions are opening clinics that promise to probe much deeper into your DNA — if you’re willing to pay hundreds or even thousands of dollars out of pocket to learn about disease risks that may be lurking in your genes.
Source Institute of Molecular Biotechnology (IMBA) Using an unbiased labeling technique, mathematical modeling, and single cell profiling to trace the footsteps of stem cells and their daughters, researchers at the University of Cambridge (UK), DGIST (S.Korea), and IMBA (Institute of Molecular Biotechnology of the Austrian Academy of Sciences, Austria) have confirmed that two populations of
Source https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.026#utm_source=ETOC&utm_medium=email&utm_campaign=hum Corneal diseases are among the most prevalent causes of blindness worldwide. The transparency and clarity of the cornea are guaranteed by a delicate physiological, anatomic, and functional balance. For this reason, all the disorders, including those of genetic origin, that compromise this state of harmony can lead to opacity and eventually vision loss.
Source The Lancet. Oncology, ISSN: 1474-5488, Vol: 20, Issue: 8, Page: e443-e451 Chimeric antigen receptor (CAR) T-cell therapy is one of the most remarkable advances in cancer therapy in the last several decades. More than 300 adoptive T-cell therapy trials are ongoing, which is a testament to the early success and hope engendered by this
New research has observed that the benefits of CAR-T cell immunotherapies – for the treatment of diseases such as pediatric acute lymphoblastic leukemia and diffuse large B-cell lymphoma – may be offset by their delayed delivery to patients. The social value of a therapy balances manufacturer costs and profits versus a patient’s improvement in quality-adjusted
The vision is ambitious : Enroll 1 million people in the NIH’s All of Us Research program, whose goal is to make personalized medicine a reality for everyone. That means ensuring that more than half of participants come from communities historically underrepresented in biomedical research. A year after its launch, 230,000 people have joined, 80% of
Countries that relax regulations for regenerative medicines could be causing a downward spiral in international standards, according to new research published today. Researchers warn that if just one country decides to relax regulations in the field, a heightened sense of competition can spur others to do the same. It’s unclear whether this deregulation best serves
Nanotechnology developed at Rutgers University-New Brunswick could boost research on stem cell transplantation, which may help people with Alzheimer’s disease, Parkinson’s disease, other neurodegenerative diseases and central nervous system injuries. The nanotechnology platform, which uses special tiny rods for sensing, allows researchers to confirm the identity of human stem cell fates and their biomarkers, or
Receptor type protein tyrosine phosphatase-sigma (PTPσ) is primarily expressed by adult neurons and regulates neural regeneration. Researchers at UCLA recently discovered that PTPσ is also expressed by hematopoietic stem cells (HSCs) and describe small molecule inhibitors of PTPσ that promote HSC regeneration in vivo. Systemic administration of the PTPσ inhibitor, DJ001, or its analog, to
