One of the most common causes of adverse neurological disabilities in newborns is neonatal hypoxic-ischemic encephalopathy. Therapeutic hypothermia (TH) is the standard therapy, but it is not efficient in all cases. Cannabinoids have raised interest as therapeutics as they are neuroprotective.
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
Scientists at Massachusetts General Hospital have linked the risk of heart failure during pregnancy and senescence proteins produced by placental aging, which could clarify how peripartum cardiomyopathy (PPCM) is triggered and opens the door to the development of cardiac function therapies in late pregnancy.
Organoids are 3D models created from human stem cells and resemble fetal tissues. In an article published in Nature Medicine on March 4, 2024, researchers from University College London provided details on the possibility of generating organoids from epithelial cells collected from amniotic fluid without terminating the pregnancy.
Neurenati Therapeutics has closed its seed funding round, securing CA$1.2 million (US$884,000) to advance development of therapies for various rare diseases, including pediatric conditions.
Researchers from Texas A&M University System have detailed the development and characterization of a novel pediatric rat model of organophosphate (OP)-induced status epilepticus (SE).
Listening to conversations between a mother and her unborn child on the cellular level could inform how the pregnancy is going and prevent complications. Three scientific groups from the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), in collaboration with Wayne State University and Michigan State University, have used single-cell RNA sequencing techniques to decipher these words, identify the cellular language of these interactions in the placenta and establish a cellular atlas.
Bloomsbury Genetic Therapies Ltd. has announced it received orphan drug designations from the FDA and the European Commission for BGT-INAD, an investigational gene therapy for the treatment of infantile neuroaxonal dystrophy (INAD).
It seems unlikely that American poet and civil rights activist Maya Angelou spent much time thinking about translational research. But two quotes of hers capture the essence of the interplay between bench and bedside: “I did then what I knew how to do. Now that I know better, I do better” and “I’ve learned that I still have a lot to learn.” At the 2023 Annual Congress of the European Academy of Neurology, Mary Reilly described the relationship between bench and bedside as “a continuous circle of translation,” with each cycle beginning with patients and their needs.
Although advances in chemotherapy have dramatically improved outcomes for children with leukemia, patients with high-risk and aggressive cancers require intense drug regimens that push safety limits. Researchers have formulated an antibody guidance system in mice that could empower chemotherapy against childhood leukemias while minimizing drug toxicity.
RSS
