Macrophages derived from human induced pluripotent stem cells (iPSCs) have the potential
to enable the development of cell-based therapies for numerous disease conditions. We …

The β-thalassemias are a group of hereditary hematological diseases caused by over 300
mutations of the adult β-globin gene. Together with sickle cell anemia, thalassemia …

Summary Interleukin-3 (IL-3) is capable of supporting the proliferation of a broad range of
hematopoietic cell types, whereas granulocyte colony-stimulating factor (G-CSF) and …

A major reason for the current reproducibility crisis in the life sciences is the poor
implementation of quality control measures and reporting standards. Improvement is …

Inborn errors of human IFN-γ-dependent macrophagic immunity underlie mycobacterial
diseases, whereas inborn errors of IFN-α/β-dependent intrinsic immunity underlie viral …

Epigenetic silencing of transgene expression represents a major obstacle for the efficient
genetic modification of multipotent and pluripotent stem cells. We and others have …

Despite developing neurosurgical procedures, few treatment options have achieved
functional recovery from traumatic brain injury (TBI). Neural stem/progenitor cells (NS/PCs) …

Induced pluripotent stem cells (iPSCs) offer a promising platform to model early embryonic
developmental processes, to create disease models that can be evaluated by drug screens …

Correction of patient-specific induced pluripotent stem cells (iPSC) upon gene delivery
through retroviral vectors offers new treatment perspectives for monogenetic diseases. Gene …

Targeted integration into a genomic safe harbor, such as the AAVS1 locus on chromosome
19, promises predictable transgene expression and reduces the risk of insertional …