Tag: <span>Gene Therapy</span>

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A French startup wants to decentralize cell and gene therapy manufacturing, making it ‘the size of a book’

Lei Lei Wu News Reporter Jérémie Laurent stumbled into biology. While studying aerospace engineering, all of Laurent’s classes were about math, so he began taking biology classes as an escape. “I just fell in love with biology,” he said. As a PhD student, he started a project at the intersection of engineering and biology —...

Liver-targeted non-viral gene therapy gives hope to hemophiliacs
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Liver-targeted non-viral gene therapy gives hope to hemophiliacs

By Paul McClure June 08, 2023 Researchers have used a non-viral approach to gene therapy, injecting DNA directly into the liver as a way of treating hemophilia Depositphotos As a way of treating hemophilia, Japanese researchers have, for the first time, delivered gene therapy directly into the livers of baboons without using a viral carrier. The study’s...

Sneaking past the blood-brain barrier to usher in the age of gene therapy for neurodegenerative disease
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Sneaking past the blood-brain barrier to usher in the age of gene therapy for neurodegenerative disease

by Justin Jackson, Medical Xpress Initial BBB openings in NHPs targeting brain regions relevant to PD. Successful BBB openings were demonstrated in two monkeys (M1/M2) by delivery of an MR contrast agent (Gd) that does not normally extravasate in the brain. Openings were achieved in the targeted regions with relative accuracy. (A) Axial contrast-enhanced T1-weighted...

New gene therapy lowers eye pressure to treat glaucoma
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New gene therapy lowers eye pressure to treat glaucoma

By Paul McClure April 20, 2023 Researchers have used gene therapy to develop a treatment for the damaging high intraocular pressure that is common in glaucoma Depositphotos The high eye pressure seen in glaucoma slowly leads to blindness. For some, the first-line treatment, eye drops, doesn’t work. Researchers have used gene therapy to develop a promising new...

Improving gene therapy with tiny bubbles
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Improving gene therapy with tiny bubbles

CASE WESTERN RESERVE UNIVERSITY IMAGE: CWRU RESEARCHERS AGATA EXNER, LEFT, AND PINUNTA “PETCH” NITTAYACHARN CREDIT: CASE WESTERN RESERVE UNIVERSITY CLEVELAND—Beyond vaccines, mRNA offers immense potential to fight disease, but targeting the genetic material to specific diseased cells is challenging—requiring a new method.  To meet this need, researchers at the Case Western Reserve University School of...

Early results of gene therapy trial for ‘childhood dementia’ show promise
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Early results of gene therapy trial for ‘childhood dementia’ show promise

by Michael Addelman, University of Manchester Credit: Pixabay/CC0 Public Domain Researchers will tell an international conference today (Feb. 24) that an investigational gene therapy for Sanfilippo syndrome—which leads to a form of childhood dementia—has shown promising early results in a proof-of-concept study. It found four out of five patients diagnosed with Sanfilippo have continued to gain cognitive skills...

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Hemophilia A Gene Therapy Durable at 2 Years; Under FDA Review

M. Alexander Otto, PA, MMS February 22, 2023 Clinical data have been published for a gene therapy for hemophilia A that is approaching the market — valoctocogene roxaparvovec (Roctavian), which is currently under review by the US Food and Drug Administration. Hemophilia A (a deficiency of clotting Factor X) is the most common form of the disease, accounting for...

Second generation gene therapy for alpha 1-antitrypsin deficiency
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Second generation gene therapy for alpha 1-antitrypsin deficiency

MARY ANN LIEBERT, INC./GENETIC ENGINEERING NEWS IMAGE: HUMAN GENE THERAPY CREDIT: MARY ANN LIEBERT, INC., PUBLISHERS Researchers report on the safety of a gene therapy to treat the common autosomal recessive hereditary disorder alpha 1-antitrypsin (AAT) deficiency in a new article in the peer-reviewed journal Human Gene Therapy. In ATT deficiency, neutrophil proteases destroy the...