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Ipsc

All articles tagged with #ipsc

iPSC-derived stem cells show strong heart-function gains in a Chinese trial
science1 month ago

iPSC-derived stem cells show strong heart-function gains in a Chinese trial

In a small Chinese study, 10 patients with severe heart failure received injections of their own induced pluripotent stem cells differentiated into heart muscle cells. Over 12 months, 9 of the 10 showed significant improvements in cardiac function and six-minute walk distance compared with a control group that underwent bypass surgery; one control patient died eight months after surgery. Some patients experienced temporary tachycardia, but no severe adverse events were reported. Researchers say longer, larger trials are needed to assess long-term safety and benefit.

Reproducible 3D human brain tissue model reveals mature microglia and Alzheimer's disease hallmarks
science2 months ago

Reproducible 3D human brain tissue model reveals mature microglia and Alzheimer's disease hallmarks

Researchers developed a reproducible iPSC-derived 3D cortical brain tissue model (3BTM) containing neurons, astrocytes and microglia that remains postmitotic and viable for months, forms brain-like ECM, and shows mature cell phenotypes. Microglia in 3BTMs display homeostatic features and in vitro–like in vivo responses. When engineered with APP mutations, 3BTMs recapitulate AD hallmarks—amyloid deposition, increased phospho-tau, and neuroinflammation—and exhibit disease-associated microglial signatures. Adding exogenous Aβ42 forms plaque-like structures, while treatment with aducanumab reduces Aβ load and enhances microglial activity, validating a translational drug-testing platform. Compared with 2D cultures, 3BTMs show greater maturation and network functionality across multiple iPSC lines, enabling scalable studies of brain physiology, disease mechanisms, and therapeutic responses.

Lab-grown dopamine cells aim to reboot movement in Parkinson’s patients
health-and-medicine7 months ago

Lab-grown dopamine cells aim to reboot movement in Parkinson’s patients

Researchers are testing implanted induced pluripotent stem cells engineered to become dopamine-producing neurons in the brains of Parkinson’s patients in a Phase 1 trial. Delivered via MRI-guided surgery into the basal ganglia, the goal is to restore dopamine production, improve motor function, and slow disease progression. The 12-person study (RNDP-001) is monitored for 12–15 months with long-term follow-up planned for up to five years to assess safety (e.g., dyskinesia, infection) and efficacy, and it has FDA fast-track designation.

Diverse autism mutations converge on a shared chromatin-regulation network in stem-cell–derived human cortex
science8 months ago

Diverse autism mutations converge on a shared chromatin-regulation network in stem-cell–derived human cortex

A large hiPSC-based study generated cortical organoids from eight ASD-risk mutations, idiopathic ASD, and controls, profiling gene expression across 25–100 days. Early mutation-specific changes give way to convergent transcriptional and chromatin-regulatory disruptions enriched for ASD risk genes, including SWI–SNF components. CRISPRi validation supports key regulators driving this convergent network, suggesting that diverse genetic risks in ASD propagate through shared transcriptional pathways that affect early neurodevelopment, while idiopathic cases show less convergence.