Tag: Gene Therapy
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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…
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Knut Steffensen Leads Karolinska ATMP Center to Bring Therapies to Patients
Moving cell and gene therapy from lab to patient care The Karolinska Institutet is establishing a dedicated ATMP Center to accelerate the development of advanced therapy medicinal products (ATMPs) and to close the gaps between discovery, manufacturing, and clinical delivery. Led by Knut Steffensen, the center aims to function as a “virtual hub” where researchers,…
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ATMP Centre at Karolinska: Bridging Cell & Gene Therapy
From Lab to Patient: The Mission of Karolinska ATMP Centre Knut Steffensen leads a bold effort to move cell and gene therapies from research benches to patient care. The ATMP Centre at Karolinska Institutet aims to shorten the distance between discovery, manufacturing, and clinical application, turning a once sci fi dream into everyday medicine. The…
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Knut Steffensen Drives Cell and Gene Therapy From Lab to Patients at Karolinska
From Lab to Patient: Turning Cell and Gene Therapy into Real Treatments Knut Steffensen is taking on the ambitious task of steering cell- and gene-based therapies from the research bench into routine patient care at Karolinska. As head of the university’s ATMP-Centre, he faces the challenge of coordinating the many moving parts required to translate…
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Huntington’s disease gene therapy offers hopeful prospects, questions
Promising but preliminary: what the results suggest A potential breakthrough in Huntington’s disease treatment has sparked cautious optimism within the community. The European biotech firm uniQure reported top-line findings from a small, early-stage trial of AMT-130, a gene therapy designed to address the genetic error behind Huntington’s disease. According to the company, those receiving the…
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Promising Huntington’s disease gene therapy slows progression, but questions remain
What the early results show News from a European biotech company on a new gene therapy has sparked guarded optimism in the Huntington’s disease community. The approach, delivered during brain surgery, targets the genetic error that drives the condition. In a small, preliminary trial involving roughly 30 participants, the highest dose of the treatment appeared…
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Huntington’s Disease Gene Therapy: Promise Amid Unanswered Questions
New Hope in Huntington’s Disease Gene Therapy A European biotech company, uniQure, has released preliminary results from an experimental gene therapy (AMT-130) aimed at Huntington’s disease. The data suggest the therapy could slow disease progression by about 75% over three years in patients receiving the highest dose. The treatment is delivered directly into the brain…
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Positive Results from Gene Therapy Trial for Huntington’s Disease
Introduction to Huntington’s Disease and Gene Therapy Huntington’s disease (HD) is a debilitating, inherited neurodegenerative disorder affecting approximately 75,000 individuals across the United States, Europe, and the United Kingdom. Characterized by progressive physical and mental deterioration, this condition arises from a mutation in the huntingtin gene, which results in the accumulation of toxic proteins in…
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Breakthrough Results in Gene Therapy for Huntington’s Disease
Groundbreaking Findings from uniQure’s Gene Therapy Trial uniQure N.V. has recently announced significant results from its Phase I/II clinical trial of AMT-130, a pioneering gene therapy aimed at treating Huntington’s disease. This pivotal study reveals a remarkable 75% slowdown in disease progression among patients treated with the high dose of AMT-130 over a 36-month period…
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Positive Gene Therapy Breakthrough for Huntington’s Disease
Introduction to Huntington’s Disease and Gene Therapy Huntington’s disease is a rare yet devastating neurodegenerative disorder affecting around 75,000 individuals across the US, EU, and UK. Characterized by progressive physical and mental decline, it stems from a mutation in the huntingtin gene, which leads to the accumulation of toxic proteins in the brain. Currently, there…