by LAURA UNGAR and FREIDA FRISARO Dr. Alfonso Sabater, checks Antonio Vento Carvajal’s eye under a blue light after applying a stain to check to see if more ulcers had developed, before a gene therapy treatment, Thursday, July 6, 2023, at University of Miami Health System’s Bascom Palmer Eye Institute in Miami. Antonio was born...
Liam Davenport July 13, 2023 An unapproved gene therapy for Leber hereditary optic neuropathy (LHON) led to a marked improvement in the eyesight of patients with a severe, progressive form of the disease who received the therapy as part of an early access program. Results of a study of more than 60 patients who received lenadogene nolparvovec...
AARHUS UNIVERSITY IMAGE: A NEW STUDY LED BY PROFESSOR YONGLUN LUO FROM THE DEPARTMENT OG BIOMEDICINE AT AARHUS UNIVERSITY SHOWS THAT ARTIFICIAL INTELLIGENCE (AI) MAY HOLD THE KEY TO A SHARPER AND MORE PRECISE DNA SCISSOR. THIS DISCOVERY MAY BE A BREAK THROUGH FOR MORE EFFICIENT GENE THERAPY. CREDIT: LINE RØNN, HEALTH AARHUS UNIVERSITY Artificial...
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 —...
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...
Reviewed by Lily Ramsey, LLMMay 26 2023 By 2050, one in 10 individuals are expected to live with some form of hearing loss. Of the hundreds of millions of cases of hearing loss affecting individuals worldwide, genetic hearing loss is often the most difficult to treat. While hearing aids and cochlear implants offer limited relief, no...
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...
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...
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...
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...
