Scientists have identified a way to "rescue" muscle cells that have genetically mutated, paving the way to a possible new treatment for rare childhood illness such as Duchenne Muscular Dystrophy (DMD).
Researchers from Indiana University have identified key genetic changes in the interstitial kidney tissue of people with diabetes, a discovery that signifies the potential for a revolutionary new genetic approach to the treatment of kidney disease. They will contribute their findings to the Kidney Precision Medicine Project's (KPMP) "cell atlas," a set of maps used to classify and locate different cell types and structures within the kidney.
MUSC Hollings Cancer Center researcher Yongxia Wu, Ph.D., identified a new target molecule in the fight against graft-versus-host disease.
A new study initiated by Wyss Core Faculty member George Church's Synthetic Biology team at Harvard's Wyss Institute for Biologically Inspired Engineering, and driven by a collaboration with Google Research has applied a computational deep learning approach to design highly diverse capsid variants from the AAV2 serotype across DNA sequences encoding a key protein segment that plays a role in immune-recognition as well as infection of target tissues. The approach has broad implications for designing more effective and safer gene therapies.
Research in Nature Biotechnology demonstrates the use of machine learning to generate unprecedented diversity of functional AAV capsids, towards evading immune system neutralization to allow more patients to benefit from gene therapies. It is estimated that up to 50-70% of the human population have pre-existing immunity to natural forms of the AAV vectors currently used in gene therapies. Dyno Therapeutics is applying CapsidMap™ and partnering with gene therapy companies to develop next-generation AAV vectors.
An international collaboration of leading groups in gene therapy and vision science initiated by George Church's group at the Wyss Institute developed a 'coupled immunomodulation' strategy in which short inflammation-inhibiting sequences are incorporated directly into the much longer AAV genome containing therapeutic DNA sequences. In different tissues of mice, as well as ocular tissues of pigs and non-human primates, the approach showed broad anti-immunogenic potential.
A team led by Brown University researchers reprogrammed patient blood cells into stem cells to test treatments for Christianson syndrome, finding that treatment responses varied according to the mutations present.
Multicellular synthetic circuits will be a much more effective way to treat cancer.
Researchers at Children's Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently used in gene therapy trials for those conditions. The vector, an engineered vehicle for delivering functional copies of the hemoglobin gene to correct a genetic abnormality, leads to the production of more hemoglobin with a lower dose, minimizing the risk of toxic side effects.
Researchers at Washington University School of Medicine in St. Louis have engineered cartilage cells to release an anti-inflammatory drug in response to stresses such cells undergo when they are compressed during weight bearing and movement.