News-Medical.Net Gene Therapy News
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Absence of key gene causes severe hearing loss
May 8, 2021 | 01:52 amA gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.
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Nanoscope presents optical gene delivery, OCT-guided electrophysiology platforms at ARVO annual meeting
May 6, 2021 | 05:48 amNanoscope Technologies LLC, a biotechnology company developing gene therapies for treatment of retinal diseases, is featuring multiple scientific presentations highlighting its groundbreaking research on optical gene delivery for vision restoration and OCT-guided electrophysiology platforms for characterization of retinal degeneration and assessment of efficacy of cell-gene therapy at the 2021 ARVO annual (virtual) meeting, May 1-7.
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Researchers use gene therapy to prevent learning, memory loss in Alzheimer's mouse model
May 5, 2021 | 06:09 amResearchers at University of California San Diego School of Medicine, with colleagues elsewhere, have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer's disease (AD), a key step toward eventually testing the approach in humans with the neurodegenerative disease.
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New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy
May 3, 2021 | 07:43 amUT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients.
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Albumedix extends research collaboration with Cobra Biologics to optimize viral vector manufacturing
Apr 28, 2021 | 13:23 pmAlbumedix Ltd. ('Albumedix'), an emerging leader in the enablement of advanced therapies and recognized global leader in recombinant human albumin (rHA) has today announced an extension to its research collaboration with Cobra Biologics, the gene therapy division of Cognate BioServices, a Charles River Laboratories International, Inc. company.
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CRISPR/Cas technology helped revolutionize diagnostics and gene therapy
Apr 26, 2021 | 14:34 pmAnnouncing a new article publication for BIO Integration journal. In this mini review article the authors Meiyu Qiu and Pei Li from Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea summarize CRISPR/Cas-based Diagnostics and Gene Therapy.
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Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases
Apr 21, 2021 | 02:59 amA first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon.
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Gene therapy for common forms of congenital blindness shows promise to improve vision
Apr 19, 2021 | 19:43 pmA new gene therapy for one of the most common forms of congenital blindness was safe and improved patients' vision, according to initial data from a clinical trial led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.
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Experts to discuss advanced cancer cell and gene therapies at Summit 2021
Apr 15, 2021 | 06:27 amApril 29 marks a pivotal moment in cancer research when the world's brightest minds come together to discuss how to translate the success of CAR T-cell therapies for blood cancers into successful cell and gene therapies for the most complex and deadly solid tumor cancers.
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Biology of single-celled organisms reveals potential treatment target for COPD
Apr 14, 2021 | 02:47 amIn a series of experiments that began with amoebas -- single-celled organisms that extend podlike appendages to move around -- Johns Hopkins Medicine scientists say they have identified a genetic pathway that could be activated to help sweep out mucus from the lungs of people with chronic obstructive pulmonary disease a widespread lung ailment.
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