Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
Credit: CC0 Public Domain RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt ...
Tag: gene
Gene editing approach helps launch first-in-human clinical trial for rare genetic disease
Credit: Unsplash/CC0 Public Domain A collaborative effort between investigators at the National Institutes of Health's National Institute of Allergy and ...
On Heels of Positive Parkinson’s Gene Therapy Data, MeiraGTx Looks Ahead to Pivotal Test
A MeiraGTx Holdings gene therapy for Parkinson’s disease has preliminary data from a small clinical trial showing the one-time treatment ...
Differential gene expression linked to alcohol use disorder, offering new treatment possibilities
New study reveals gene expression differences in brain regions tied to addiction, highlighting pathways for innovative alcohol use disorder treatments ...
Disrupting Asxl1 gene prevents T-cell exhaustion to improve immunotherapy, researchers discover
Core epigenetic regulators in maintaining therapeutic durable multipotent Tpex populations. Credit: Science (2024). DOI: 10.1126/science.adl4492 Immunotherapy, using a patient's own ...
Study reveals key role of TRIO gene in epileptic encephalopathies
Defective migration of MGE-derived cortical INs in Trio–/– embryos. Credit: Molecular Psychiatry (2024). DOI: 10.1038/s41380-024-02742-y What are the molecular and ...
Faulty gene makes the brain too big—or too small
Credit: The American Journal of Human Genetics (2024). DOI: 10.1016/j.ajhg.2024.07.016 A gene called ZNRF3, known to be involved in cancer, ...
Pfizer Hemophilia Gene Therapy Meets Phase 3 Goals, But Its Success Matters More to Sangamo
A Pfizer gene therapy for hemophilia A reduced bleeding rates in patients with the inherited blood disorder, meeting goals of ...
Lexeo’s gene therapy for rare heart disease shows promise in trial
Lexeo Therapeutics stated Monday {that a} gene remedy designed to stop deadly cardiac issues of a genetic dysfunction known as ...
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