Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.
Researchers reviewed how somatic mutations accumulate in normal and diseased tissues, showing that natural selection can expand clones that influence disease risk, tissue adaptation, or cellular ...
The CMS Innovation Center is approaching the next negotiation cycle for the CGT Access Model. Several considerations will guide the selection of future candidate diseases and potential new model ...
The International Society for Stem Cell Research (ISSCR) is pleased to announce that Alexander Meissner, Director at the Max Planck Institute for Molecular Genetics, Germany, has been named the ...
Human pluripotent stem-cells (HPSCs)—including human embryonic stem-cells and human induced pluripotent stem-cells—are reshaping the landscape of regenerative medicine.1,2 In Parkinson's disease, ...
Morning Overview on MSN
Gene editing advances raise real prospects for future DNA upgrades
The U.S. Food and Drug Administration approved the first cell-based gene therapies for sickle cell disease, including the first-ever treatment built on CRISPR/Cas9 technology. That decision moved gene ...
"Population-scale spatial has arrived and is on center-stage at AGBT 2026," stated Christopher Mason, PhD, Professor of Physiology and Biophysics at Weill Cornell Medicine. " [Jiwoon Park] is ...
February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world's first person to receive a personalized CRISPR-based gene ...
Cystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in CTNS, the gene encoding cystinosin, a lysosomal transmembrane cystine transporter. In patients with cystinosis ...
Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy—the transplantation of living cells—and gene therapy—the use of ...
A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...
After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by the FDA. In rejecting RGX-121, the FDA raised concerns about its clinical ...
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