Gene therapies on the horizon for sickle cell disease: a clinician's perspective.

Introduction: Sickle cell disease (SCD) is a monogenic disorder that exerts several detrimental health effects on those affected, ultimately resulting in significant morbidity and early mortality. There are millions of individuals globally impacted by this disease. Research in gene therapy has been growing significantly over the past decade, now with two FDA approved products, aiming to find another cure for this complex disease.

Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models.

Ex vivo resting culture is a standard procedure following genome editing in hematopoietic stem and progenitor cells (HSPCs). However, prolonged culture may critically affect cell viability and stem cell function. We investigated whether varying durations of culture resting times impact the engraftment efficiency of human CD34+ HSPCs edited at the BCL11A enhancer, a key regulator in the expression of fetal hemoglobin.

Multi-Generational Review of Oncologic Tumors in a Family With TP53 Mutation Presenting With a Pediatric Patient With Osteosarcoma and Lung Acinar Adenocarcinoma.

TP53 mutation, Li-Fraumeni syndrome (LFS), is a syndrome that leads to a hereditary cancer predisposition. Here we describe the case of a 13-year-old male who presented with osteosarcoma, family history of LFS, who developed a second primary tumor of the lung. No other similar cases have been reported.