His father's voice, the sounds of passing cars and scissors clipping his hair: An 11-year-old boy is hearing for the first time in his life after receiving a breakthrough gene therapy.
Jan 23, 2024
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The Ultrasound and Elasticity Imaging Laboratory, led by Elisa Konofagou, Robert and Margaret Hariri Professor of Biomedical Engineering, develops novel, ultrasound-based techniques for both imaging and therapeutic applications.
Sep 13, 2023
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An international team of researchers has developed a new method to deliver drugs into the inner ear. The discovery was made possible by harnessing the natural flow of fluids in the brain and employing a little understood ...
Jun 28, 2023
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In a paper titled "BBB opening with focused ultrasound in nonhuman primates and Parkinson's disease patients: Targeted AAV vector delivery and PET imaging," published in Science Advances, researchers led by the University ...
The use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV). The study, which showed that this strategy enabled gene ...
Mar 22, 2023
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The blood-brain barrier (BBB) is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain tissue, but it also keeps ...
Oct 10, 2022
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Completely dislodging a tooth from the socket is not generally considered a reversible process. However, this injury is most common in children, whose roots may not be completely developed, meaning quick reactions could save ...
Sep 16, 2021
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In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children's Hospital of Philadelphia (CHOP) have developed a "dimmer switch" system that can control levels of proteins ...
Jul 28, 2021
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In a new study published in the Cell Research, Chen-Yu Zhang's group at Nanjing University reports "In vivo self-assembled small RNA is the new generation of RNAi therapeutics."
Mar 30, 2021
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The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible ...
Oct 9, 2020
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