Researchers from the Universities of Bologna and Torino recently presented their hematopoietic stem cell gene therapy (HSC-GT) strategy based on microglia-mediated delivery using a lentiviral vector encoding a secretable, cell-penetrating CDKL5 protein (Igκ-TATk-CDKL5).
Respiratory syncytial virus (RSV) infection is responsible for 3.2 million hospital admissions and about 118,000 deaths in children under 5 years of age. The increased risk of RSV infection (and other infectious diseases) in young children is broadly attributed to their immature immune system. But researchers from University College London have published work suggesting that this is not the only reason.
The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services, has announced the teams for the THRIVE (Treating Hereditary Rare diseases with In Vivo prEcision genetic medicines) program. With a commitment of up to $160 million over 5 years, THRIVE aims to accelerate solutions for rare genetic pediatric diseases across multiple technological approaches, clinical trial designs and deployment models.
