Contents
Vol 3, Issue 97
Contents
Commentary
- Herding CATS
The National Center for Advancing Translational Sciences seems primed to polarize scientists while possibly failing to drive biomedical research and development forward.
Perspective
- A Tale of Two SCIDs
Gene therapy for severe combined immunodeficiency: Do the benefits outweigh the risks?
Research Articles
- Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency
Gene therapy results in long-term persistence of T cells in immunodeficient patients.
- Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction
Gene therapy can restore immune and metabolic function in patients with adenosine deaminase immunodeficiency.
- Potent Kinetic Stabilizers That Prevent Transthyretin-Mediated Cardiomyocyte Proteotoxicity
A high-throughput screen for transthyretin ligands that inhibit protein aggregation identifies agents that prevent amyloid formation and toxicity toward cardiomyocytes.
Editors' Choice
- PTPN22: A Risky Business
Healthy carriers of the major risk variant of PTPN22 show early signs of autoimmunity.
- A Picture Is Worth a Thousand Dollars?
Screening for lung cancer with low-dose CT scans reduces mortality, but may cost significantly more than current methods.
- Blue-CHIP Diagnostics
“Lab on a chip” technology is used for rapid diagnosis of HIV and syphilis in a resource-poor setting.
- Brings Back Memories
A neurophysiology study of prefrontal cortical neurons may advance medications that alleviate age-related decline in memory.
