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Precision BioSciences Announces New Study Published in Nature Communications Using Engineered ARCUS Nuclease to Target Mutant Mitochondrial DNA In Vivo

Published: Jun 01, 2021
June 1, 2021 11:00 UTC
Learnings to be Presented by Carlos Moraes, Ph.D., from University of Miami at UMDF’s Mitochondrial Medicine Symposium on June 4, 2021
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and
in vivo gene correction therapies with its ARCUS® genome editing platform, today announced a new paper published online in
Nature Communications that reports
preclinical results using an ARCUS nuclease to target mitochondrial DNA (mtDNA) and reduce levels of mutant mtDNA
in vivo.
The study, “Mitochondrial targeted meganuclease as a platform to eliminate mutant mtDNA in vivo” was led by Carlos T. Moraes, Ph.D., Esther Lichtenstein Professor in Neurology at the University of Miami Miller School of Medicine, with Ugne Zekonyte as first author. ....

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Precision BioSciences Announces New Study Published in Nature Communications Using Engineered ARCUS

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01-Jun-2021
Precision BioSciences Announces New Study Published in Nature Communications Using Engineered ARCUS Nuclease to Target Mutant Mitochondrial DNA In Vivo

Learnings to be Presented by Carlos Moraes, Ph.D., from University of Miami at UMDF’s Mitochondrial Medicine Symposium on June 4, 2021
DURHAM, N.C. (BUSINESS WIRE) Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and
in vivo gene correction therapies with its ARCUS® genome editing platform, today announced a new paper published online in
Nature Communications that reports
preclinical results using an ARCUS nuclease to target mitochondrial DNA (mtDNA) and reduce levels of mutant mtDNA ....

United States , University Of Miami , Ugne Zekonyte , Alex Kelly , Carlos Moraes , Carlost Moraes , Derek Jantz , Precision Biosciences Inc , Nature Communications , Virtual Meet The Scientific Program Faculty On , United Mitochondrial Disease Foundation Medicine , University Of Miami Miller School Medicine , Medicine Symposium , Precision Biosciences , Esther Lichtenstein Professor , Miami Miller School , Chief Scientific Officer , United Mitochondrial Disease Foundation , Mitochondrial Medicine , Scientific Program , Private Securities Litigation Reform Act , Quarterly Report , Recent Advances , Annu Rev Genomics Hum Genet , ஒன்றுபட்டது மாநிலங்களில் , பல்கலைக்கழகம் ஆஃப் மியாமி ,

New drug molecules hold promise for treating fatal child disease

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Scientists have identified a way to rescue muscle cells that have genetically mutated, paving the way to a possible new treatment for rare childhood illness such as Duchenne Muscular Dystrophy (DMD).
The study, led by the Universities of Exeter and Nottingham, is published in the
Proceedings of the National Academies of Sciences, USA. The research used novel drugs being developed at the University of Exeter, which metabolically reprogram the cellular energy production centres in muscle cells, by providing them with a fuel source to generate metabolic energy.
DMD is a genetic condition caused by a mutation in a gene called dystrophin which results in progressive irreversible muscular degeneration and weakening. Its symptoms include muscle atrophy leading to a loss of the ability to walk in children for which there is no known cure. Currently, the condition is treated with steroids, such as prednisone, but they can stop working and side-effects are com ....

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Study paves the way to a potential new treatment for rare terminal childhood illness

Study paves the way to a potential new treatment for rare terminal childhood illness
Scientists at the University of Exeter have identified a way to rescue cells that have genetically mutated, paving the way to a possible new treatment for rare terminal childhood illness such as mitochondrial disease.
The research, funded by the United Mitochondrial Disease Foundation in the USA, was led by Professors Matt Whiteman and Tim Etheridge. In the study, published in the
Journal of Inherited Metabolic Disease, the team used novel drugs being developed at the University of Exeter, which metabolically reprogramme mitochondria - the cellular energy production centres in cells, by providing them with an alternative fuel source to generate metabolic energy in the form of minute quantities of hydrogen sulfide. ....

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