News
Gene therapy for cardiac and vascular disease
2+ hour, 25+ min ago (154+ words) Nature Thank you for visiting nature. com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet…...
Pro QR Therapeutics Unveils New RNA Editing Programs; AX-0810 Phase 1 Target Data Due This Quarter
7+ hour, 11+ min ago (143+ words) In the Q&A, L'pez-L'pez clarified that the two-fold threshold for total bile acids would be evaluated "both compared to baseline as well as compared to placebo." Management also emphasized the trial remains double-blind and placebo-controlled, and the company has…...
Science history: Doctor identifies transmissible proteins
5+ hour, 52+ min ago (482+ words) Prion diseases, such as "mad cow," are caused by transmissible proteins that were identified in the 1980s. Date: April 9, 1982 Who: Dr. Stanley Prusiner On April 9, 1982, a doctor at the University of California, San Francisco, published a paper in the journal Science…...
Hidden numbers: Genetic cholesterol conditions raise heart disease risk
10+ hour, 36+ min ago (418+ words) A Maine woman hopes others will take the time to learn their risks, especially when it comes to conditions you can't see For many people, high cholesterol is something that develops over time. But for others, Carolyn Lebeis, it's something…...
Yong-Hui Jiang: Unlocking Genetic Mysteries to Treat Brain Disorders
23+ hour, 7+ min ago (1639+ words) As a young doctor at a pediatric hospital in Shanghai, Yong-Hui Jiang, MD, Ph D, looked into the faces of parents and saw desperation. Working in a clinic for children born with Down syndrome and significant cognitive delay, Jiang could…...
'It's saving his life': FDA approves new drug for rare disease, helping Kansas family
1+ day, 3+ hour ago (737+ words) Atlas Neuerburg, 4, was diagnosed with Hunter syndrome at 18 months, and his parents have been advocating for FDA approval of the treatment ever since. OVERLAND PARK, Kan. (KSHB) -- The FDA gave accelerated approval for a groundbreaking drug that helps treat Hunter…...
Stem cell opportunities expand " and so does GBI
23+ hour, 54+ min ago (425+ words) Jesse Mc Cool, the recently named president of GBI Biomanufacturing, is new to living in Florida " though there have been regular visits as a son is a University of Florida "Gator." Still, Mc Cool's expertise in the manufacture of biologics…...
Base Editing Shows Early Promise for Treating Beta Thalassemia
1+ day, 52+ min ago (560+ words) The Chinese biotech Correct Sequence Therapeutics, also known as Correctseq, reports good results from a Phase I study of its technology involving editing a person's hematopoietic stem cells to treat beta thalassemia. The trial, published in Nature, included five patients…...
Immune-Stealth DNA Enables Safer, Large-Scale Genome Writing
1+ day, 2+ hour ago (187+ words) A new genome-editing strategy could sidestep one of the field's biggest bottlenecks: the immune response triggered by double-stranded DNA (ds DNA). Researchers have developed "INSTALL," a system that pairs recombinases with circular single-stranded DNA (css DNA) donors to enable kilobase-scale…...
Genflow reports sustained safety and efficacy three months after gene therapy dosing in dog trial
1+ day, 7+ hour ago (317+ words) This record is published on behalf of Genflow Biosciences Ltd, a paid client of Proactive In exchange for publishing services rendered by the Company on behalf of Genflow Biosciences Ltd named herein, including the promotion by the Company of Genflow…...