Parkinson disease (PD) can be caused in different cases by a number of complex factors, including genetics, lifestyle, environmental factors, and other factors that may not...
Researchers from the University of Missouri School of Medicine found that a targeted gene therapy may make acute myeloid leukemia (AML) more sensitive to chemotherapy, while...
Findings published in Molecular Therapy examined the clinical immunogenicity outcomes of valoctocogene roxaparvovec, an adeno-associated virus serotype 5 (AAV5)–vectored gene therapy, in adults with severe hemophilia...
TOPLINE: A single dose of EDIT-101, a CRISPR-associated protein 9 (Cas9)-based gene-editing therapy, appears safe and improves vision and sensitivity to light in patients with retinal...
Hearing loss is one of the most prevalent disorders and it is estimated that over one billion people suffer from hearing loss and approximately one-two children...
A novel nonviral nanovector is a highly efficient delivery system for dual–messenger RNA (mRNA) immunogene therapy against colon cancer, suggest the results of recent mouse-model research....
For most of us, the joys of hearing a loved one’s voice or the simple pleasure of listening to music is a given. But for five...
In a recent study published in Nature Medicine, researchers evaluated the safety and effectiveness of binaural adeno-associated virus 1 (AAV1)-human otoferlin (hOTOF) therapy in five children...
Gene-based therapy may be an answer to long-sought treatment for the rare disease Timothy syndrome (TS), suggest new study findings published in Nature.1 The proof-of-concept study...