Gene Therapy – Newsemia

Plant-based relatives of cholesterol could boost gene-infused nanoparticle delivery

NewsemiaFebruary 20, 2020

by NewsemiaFebruary 20, 2020025

Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol because their shape and structure help the genes get where...

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Gene therapy approach could pave the way for treatment of Stargardt disease

NewsemiaFebruary 8, 2020

by NewsemiaFebruary 8, 2020032

A leading Professor of Ophthalmology at the University of Oxford has shared the details of his research which could pave the way for a new...

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New gene therapy improves vision in blind mice

NewsemiaFebruary 7, 2020

by NewsemiaFebruary 7, 202004

Mice born blind have shown significant improvement in vision after undergoing a new gene therapy developed by a team of Japanese scientists. Source link...

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Rejuvenate Bio secures license to commercialize gene therapy for treating age-related diseases in dogs

NewsemiaFebruary 7, 2020

by NewsemiaFebruary 7, 2020023

Rejuvenate Bio has secured a license to commercialize a gene therapy technology developed at the Wyss Institute to treat multiple age-related diseases in dogs, which...

Neurology

Maybe Memorizing the Krebs Cycle Was Worthwhile After All

NewsemiaFebruary 7, 2020

by NewsemiaFebruary 7, 202004

Mitochondrial disorders are often difficult to diagnose and treat due to their multisystem nature. As scientists better understand mitochondrial function, effective treatments are beginning to...

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SITESEEKER to be used for Otsuka’s therapeutics discovery programs

NewsemiaJanuary 21, 2020

by NewsemiaJanuary 21, 2020013

PhoreMost will deploy its in-house expertise and next-generation phenotypic screening platform, SITESEEKER, in disease-relevant pathways nominated by Otsuka. Source link...

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Scientists discover a new way to combine coding languages into single "bilingual" molecule

NewsemiaJanuary 10, 2020

by NewsemiaJanuary 10, 2020016

The nucleic acids of DNA encode genetic information, while the amino acids of proteins contain the code to turn that information into structures and functions....

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Gene Therapy Breakthrough Could Treat Kidney Diseases

NewsemiaJanuary 7, 2020

by NewsemiaJanuary 7, 2020016

A new study has rekindled the hope kidney diseases can be treated with gene therapy. Source link...

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Study shows safety, long-term beneficial impact of gene therapy for patients with Hemophilia A

NewsemiaJanuary 6, 2020

by NewsemiaJanuary 6, 2020018

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in...

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Study shows the feasibility of using gene therapy to treat chronic traumatic encephalopathy

NewsemiaJanuary 4, 2020

by NewsemiaJanuary 4, 2020015

A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy. Source link...

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Study uncovers a previously unknown subtype of muscle stem cells

NewsemiaDecember 21, 2019

by NewsemiaDecember 21, 201903

Scientists have discovered a new subtype of muscle stem cells. These cells have the ability to build and regenerate new muscles, making them interesting targets...

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Neuroscientists develop new method for uniquely targeted gene therapy

NewsemiaDecember 14, 2019

by NewsemiaDecember 14, 201900

Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact...

Neurology

University Of Iowa wins $18 million grant to continue study on Huntington's disease

NewsemiaDecember 14, 2019

by NewsemiaDecember 14, 201901

Peg Nopoulos, MD, chair of psychiatry and professor of psychiatry, neurology, and pediatrics in the University of Iowa Carver College of Medicine, has received a...

Neurology

SUNFISH: Risdiplam Meets Primary Endpoint in SMA

NewsemiaNovember 13, 2019

by NewsemiaNovember 13, 201902

Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in...

Pediatrics

Beverly Davidson, PhD, Elected to the National Academy of Medicine

NewsemiaOctober 22, 2019

by NewsemiaOctober 22, 201900

Beverly L. Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia, has been elected to...

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Faster, cheaper, better: improved gene therapy tool production

NewsemiaSeptember 23, 2019

by NewsemiaSeptember 23, 201903

The heart of modern therapy is increasingly personalized and targeted at specific cellular or genetic deficits. This requires gene therapy, in many cases. A new...

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New gene therapy helps stroke patients develop new neurons

NewsemiaSeptember 12, 2019

by NewsemiaSeptember 12, 201900

Ischemic stroke is a condition in which parts of the brain lose their blood supply, causing nerve damage. Once brain tissue suffers irreparable harm, the...

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Nanocapsules packed with gene-editing tools could be promising platform for many gene therapies

NewsemiaSeptember 10, 2019

by NewsemiaSeptember 10, 201900

New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method...