CAR‑T, CRISPR, in vivo delivery, manufacturing, and long‑term follow‑up.
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The therapy was designed and manufactured in roughly six months. But those six months were possible only because decades of scientific work had already laid the groundwork before KJ was born. Before anyone knew what it was for When medicine…
A new review charts how in vivo CAR-T engineering could transform customized living drugs into scalable, off-the-shelf genetic medicines.
CRISPR Cas9 NHS availability follows a rigorous clinical pipeline governed by regulatory assessments, with treatments evaluated strictly on evidence-based safety and therapeutic value. While standard NHS rollout requires formal approval pathways before reaching hospital wards, gene-editing interventions represent a major evolution…
Romy Kerschbaumer was 48 when her life changed forever after she started getting symptoms of Huntington's disease. She was forced to medically retire from the job she "absolutely loved" setting IT strategy for a government department in Jersey. Huntington's disease relentlessly…
Bone and soft tissue sarcomas remain challenging to diagnose and monitor because of their rarity, molecular heterogeneity, low circulating biomarker abundance, and the limited sensitivity of conventional surveillance for microscopic disease. This narrative review synthesizes current evidence on liquid biopsy…
Legend Biotech announced that the U.S. Food and Drug Administration (FDA) had cleared its investigational new drug (IND) application for LB2501, an investigational in vivo CAR-T therapy for relapsed or refractory B-cell malignancies, including B-cell non-Hodgkin lymphoma. According to the company’s announcement,…
CRISPR-based synthetic lethal screening has identified the acetyltransferases p300 and CBP as essential chromatin partners for YAP-driven transcription in GNAQ-mutant uveal melanoma, pointing to a new therapeutic target for this hard-to-treat eye cancer. Uveal melanoma, the most common primary cancer of…
Drug-resistant tuberculosis remains one of the most stubborn obstacles in global infectious disease control, and a new mini review in Molecular Biology Reports argues that the CRISPR-Cas system has become the decisive research tool for understanding how Mycobacterium tuberculosis evades…
Friends of Murdoch Children’s Research Institute (MCRI) gathered at Old Mates — the haute Aussie pub co-owned by Hugh Jackman in NYC — on Thursday night. Mixing downtown cool and authentic Australian pub fare — including sausage rolls, oysters Kilpatrick and pavlova — the…
Infant neuroblastoma is one of the most clinically paradoxical cancers in medicine. Diagnosed during the first year of life, it spans a spectrum from localized tumors that can shrink or vanish entirely without treatment to aggressive metastatic disease that resists…