Immunoids: Building immunocompetent organoids from human pluripotent stem cells

Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease

Genomic and transcriptomic quality control for an autologous iPSC-derived cell therapy for Parkinson’s disease

H3K27M drives OPC stemness and intrathecal therapeutic vulnerability in brainstem glioma organoids

Clinical base editing for β-hemoglobinopathies across different genetic backgrounds

IGF2 acts downstream of Hippo signaling to regulate organ growth and restore liver regeneration in aging

Endothelialized callus organoids drive rapid regeneration of critical-size segmental long bone defects

Epi-Allele elicits compensatory expression of the non-targeted allele and prevents haploinsufficiency in dominant genetic diseases

Engineered human iPSC-derived dendritic cells dressed with tumor MHC complexes as a cancer vaccine

The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS

Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells

Flipping the fetal switch across β-hemoglobinopathies

Activins and BMPs: The yin and yang of diffuse midline gliomas

Right size, right time: The Hippo-IGF2 axis calibrates liver growth and regeneration

Silencing the mutant allele without compromising gene dosage