Tag: Gene Therapy
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Experimental gene therapy restores immune system function in ADA-SCID children
Groundbreaking therapy offers durable immune restoration for ADA-SCID An experimental gene therapy developed by researchers at UCLA, University College London (UCL) and Great Ormond Street Hospital (GOSH) has achieved durable restoration of immune system function in 59 of 62 children born with ADA-SCID, a rare and often deadly genetic immune disorder. The long-term follow-up results,…
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Long-term success for gene therapy in children with ADA-SCID: a landmark study
Groundbreaking long-term results for ADA-SCID gene therapy A multinational team led by Great Ormond Street Hospital (GOSH), University College London (UCL), and the University of California, Los Angeles (UCLA) has reported the longest follow-up to date for children treated with a gene therapy for ADA-SCID, a rare immune deficiency. The study, published in the New…
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Experimental Gene Therapy Restores Immune Function in Children with ADA-SCID
Breakthrough in ADA-SCID treatment: Gene therapy shows lasting immune restoration Researchers have reported a landmark advance in treating ADA-SCID, a life-threatening genetic immune disorder. An experimental gene therapy developed by teams at UCLA, University College London (UCL), and Great Ormond Street Hospital (GOSH) restored and sustained immune system function in 59 of 62 children born…
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Experimental Gene Therapy Restores Immune Function in Children with ADA-SCID: A Breakthrough in Immunodeficiency Treatment
Overview: A new path for ADA-SCID treatment Researchers from UCLA, University College London, and Great Ormond Street Hospital have reported a remarkable milestone in the fight against ADA-SCID, a rare and often fatal genetic immune disorder. In a long-term study published in the New England Journal of Medicine, an experimental gene therapy restored and sustained…
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Gene Therapy Shows Long-Term Success for Children with ADA-SCID
Groundbreaking Study Highlights Long-Term Success of ADA-SCID Gene Therapy A landmark international study led by researchers from Great Ormond Street Hospital (GOSH), University College London (UCL), and the University of California, Los Angeles (UCLA) shows that gene therapy can provide durable, life-changing results for children with ADA-SCID, a rare and life-threatening immune deficiency. The team…
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QIMR Berghofer set for governance overhaul after 80 years
Introduction: A major governance shift for QIMR Berghofer The Queensland government has announced a far-reaching governance overhaul for the Queensland Institute of Medical Research (QIMR) Berghofer, aiming to modernise an institution with an 80-year legacy. The reforms seek to expand access to incentive payments, strengthen accountability, and improve flexibility and commercial outcomes for one of…
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QIMR Berghofer Governance Overhaul Expands Access
Queensland Announces a Governance Overhaul for QIMR Berghofer The Queensland government has unveiled a major reform package aimed at expanding access to incentive payments and strengthening the governance framework surrounding the Queensland Institute of Medical Research (QIMR) Berghofer. The changes, described by ministers as a necessary step to improve accountability, flexibility, and commercial outcomes, mark…
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NeurologyLive Brain Games: Exploring Duchenne Muscular Dystrophy Gene Therapy
Welcome to NeurologyLive® Brain Games NeurologyLive® Brain Games returns with a focused look at neurology science through a weekly, clinician-written quiz. While the collection spans dementia, epilepsy, migraine, movement disorders, and more, this week centers on the cutting-edge topic of Duchenne muscular dystrophy (DMD) gene therapy. Each installment challenges you with three questions that blend…
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NeurologyLive Brain Games: Duchenne Gene Therapy Quiz — October 12, 2025
NeurologyLive Brain Games: A weekly neurology trivia spotlight NeurologyLive® Brain Games is a weekly quiz series designed for clinicians, researchers, and neurology enthusiasts. Fresh questions go live every Sunday morning and challenge you across a spectrum of topics—from dementia and epilepsy to stroke, sleep disorders, and the cutting edge of gene therapy. This week’s theme…
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Advancing Gene Editing: The Role of Lipid Nanoparticles in CRISPR Delivery
Introduction: Why delivery platforms matter in CRISPR medicine CRISPR-based therapies hold immense promise for treating a wide range of diseases, but their success hinges not only on the gene-editing tool itself but also on how the therapeutic payload reaches target cells. With nucleic acid–based CRISPR medicines, delivery is as crucial as the editing technology. While…