Sarepta Therapeutics (NASDAQ:SRPT), a biotechnology company specializing in precision genetic medicines for rare diseases with a market capitalization of $11.2 billion, stands at a critical juncture ...

The dengue virus (DENV) is a single-stranded RNA virus with increasing worldwide prevalence causing severe mosquito-borne viral infections. Therefore, effective strategies to prevent and treat DENV ...

Jefferies projects a peak opportunity of at least $3 billion for DYNE-101. Dyne is also running another candidate—the phosphorodiamidate morpholino oligomer DYNE-251 being tested for Duchenne muscular ...

Lines 328-334: There have been many publications describing appropriate morpholino controls. The authors need to describe their controls and show that they know that the genes they were targeting were ...

Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. Chloride ions are important inducers of salt ...

This important work advances our understanding of the contribution of tissue-resident immune cells to trained immunity phenotypes. The evidence supporting the claims is convincing, with results that ...

Marfan syndrome (MFS) is an inherited disorder that affects the connective tissues and mainly presents in the bones, eyes, and cardiovascular system, etc. Aortic pathology is the leading cause of ...

Digenic inheritance (DI) is the simplest form of inheritance for genetically complex diseases. By contrast with the thousands of reports that mutations in single genes cause human diseases, there are ...

The FDA approval of casimersen was based on the increased dystrophin production observed in patients with DMD who were treated with the drug. 1 The increased production of a functional protein may be ...

Introduction: To design effective small molecule inhibitors targeting the immune checkpoint PD-1/PD-L1 and to explore their inhibitory activity. Methods: In this paper, a total of 69 PD-1/PD-L1 ...