Vector integration and fate in the hemophilia dog liver multiple years after AAV-FVIII gene transfer.

Gene therapy using adeno-associated virus (AAV) vectors is a promising approach for the treatment of monogenic disorders. Long-term multiyear transgene expression has been demonstrated in animal models and clinical studies. Nevertheless, uncertainties remain concerning the nature of AAV vector persistence and whether there is a potential for genotoxicity.

Metabolically Active Zones Involving Fatty Acid Elongation Delineated by DESI-MSI Correlate with Pathological and Prognostic Features of Colorectal Cancer.

Colorectal cancer (CRC) is the second leading cause of cancer deaths. Despite recent advances, five-year survival rates remain largely unchanged. Desorption electrospray ionization mass spectrometry imaging (DESI) is an emerging nondestructive metabolomics-based method that retains the spatial orientation of small-molecule profiles on tissue sections, which may be validated by 'gold standard' histopathology.

Long-term follow-up of liver-directed, adeno-associated vector-mediated gene therapy in the canine model of hemophilia A.

Questions remain concerning the long-term efficacy, safety, and site(s) of transgene expression following adeno-associated vector (AAV) therapy. We report a long-term follow-up of 8 (male = 4, hemizygous, and female = 4, homozygous) dogs with severe hemophilia A treated with a single portal vein infusion of a B-domain-deleted (BDD)-canine FVIII (cFVIII) AAV vector (median dose = 1.25 × 1013 vg/kg, AAV2 = 4, AAV6 = 3, and AAV8 = 1).

The utility of image-enhanced endoscopy and Lugol's for the assessment of esophageal squamous carcinoma.

Video 1This is a case of a patient with esophageal squamous cell carcinoma who underwent endoscopic submucosal dissection and subsequent esophagectomy. The endoscopic findings using imaged endoscopy, magnification, and Lugol's are demonstrated. There is a demonstration of endoscopic and pathological correlation in addition to the emphasis on a patient-centered multidisciplinary approach to esophageal neoplasia and the diagnostic utility of endoscopic submucosal dissection.