Preclinical

1. The adeno-associated virus 5 (AAV5)-based gene therapy, valoctocogene roxaparvovec (AAV5-hFVIII-SQ), resulted in sustained factor VIII production, reduced bleeding rates,...

1. LentiGlobin gene therapy resulted in sustained production of the modified anti-sickling hemoglobin, HbAT87Q. 2. LentiGlobin led to reduced hemolysis...

1. Betibeglogene autotemcel (beti-cel) gene therapy resulted in sustained hemoglobin A with a T87Q amino acid substitution (HbAT87Q) in non-β0/β0...

1. Children of mothers who were exposed to anti-psychotics during pregnancy, compared to those never exposed, did not have an...

1. There was no difference in follow-up symptom severity between patients who received immediate or delayed antibiotics for respiratory tract...

1. Whole-genome sequencing for myeloid cancers, compared to standard cytogenic analysis, was able to more effectively risk-stratify cytogenic cases. 2....

1. Nonhuman primate vaccination with messenger RNA (mRNA)-1273 induced rapid neutralizing activity against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)....

1. An oral compound, SEP-363856 was shown to significantly reduce the total score of the Positive and Negative Symptom Scale...

1. A small number of individuals with confirmed COVID-19 were found to have viral RNA in their feces (29%) and...

1. Acute optic neuritis appears may be an appropriate condition to test neuroprotective and re-myelinating therapies after acute inflammation in...