Polypurine hairpins are used to inhibit PCSK9 expression. When cholesterol levels in the blood rise too high, a condition known as hypercholesterolemia can develop, damaging arteries and threatening ...
Study results published in The New England Journal of Medicine show the therapy significantly improved hearing in nearly a ...
Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...
Sana Biotechnology, Inc. is working on a gene therapy to cure Type 1 diabetes. Click here to find out why SANA stock is a ...
Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...
Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...
Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.
The gene therapy for neurological diseases market is poised for growth due to rising neurological disorder prevalence, demand ...
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