Bothnia dystrophy is a form of hereditary blindness, prevalent in the region Västerbotten in Sweden. A new study at Karolinska Institutet published in Nature Communications shows that gene therapy can improve vision in patients ...
Sep 10, 2024
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Adeno-associated virus (AAV)-based viral vectors used in human gene therapy can trigger innate immune pathways, leading to the initiation of adaptive immune responses. A new review article published in the journal Human Gene ...
Jul 10, 2024
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An innovative tool for the targeted modification of gene activity in heart muscle cells could establish itself as a standard method for research into cardiovascular diseases.
May 14, 2024
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Scientists often label cells with proteins that glow, allowing them to track the growth of a tumor, or measure changes in gene expression that occur as cells differentiate.
May 10, 2024
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A clinical trial led by researchers at Washington University School of Medicine in St. Louis has shown that a T cell immunotherapy—in which the patients' own T cells are genetically modified to attack and kill cancer cells—is ...
Mar 28, 2024
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A research team led by the University of California, Irvine has created 20 new recombinant rabies viral vectors for neural circuit mapping that offer a range of significant advantages over existing tools, including the ability ...
Feb 14, 2024
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A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of ...
Jan 30, 2024
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Rice University researchers tested the safety and feasibility of gene delivery to multiple brain regions using a noninvasive, ultrasound-based technique in rodents, and their findings suggest that the efficiency of gene delivery ...
Sep 27, 2023
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Gene therapy might offer a one-time, sustained treatment for patients with serious alcohol addiction, also called alcohol use disorder, according to a new study led by a researcher at The Ohio State University Wexner Medical ...
Aug 14, 2023
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The lone volunteer in a gene-editing study targeting a rare form of Duchenne muscular dystrophy likely died after having a reaction to the virus that delivered the therapy in his body, researchers concluded in an early study.
Jun 1, 2023
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