Abstract: The present invention is related to use of recombinant lentiviral vectors containing a therapeutic gene of interest fused in-frame with an intercellular trafficking gene for the global delivery of therapeutic proteins in nondividing cells.

Abstract: Fluorogenic substrates and compositions containing the same are useful for detecting enzymatic activity within intact cells. The fluorogenic substrates are lysosomotropic derivatives of 2,3-dicyano-hydroquinone (DCH). These derivatives may be employed for detecting lysosomal enzymatic activity within intact cells using fluorescent microscopy and cytometry.

Abstract: An artificial substrate consisting of a 2-alkanoylamino-4-nitrophenyl phosphorylcholine hydroxide is used for determining the sphingomyelinase activity in an extract of human cells or tissues by incubating the substrate together with the extract, whereby sphingomyelinase-catalyzed hydrolysis of the substrate into the corresponding 2-alkanoylamino-4-nitrophenol and phosphorylcholine takes place in an amount proportional to the sphingomyelinase activity in the extract. Alkalinization of the 2-alkanoylamino-4-nitrophenol thereby produced to convert it into the alkali salt results in the development of a bright yellow color whose intensity is proportional to the amount of the substrate that has been hydrolyzed. Measurement of the intensity of the yellow color, for example, by means of a simple colorimeter or photometer, provides an accurate indication of the sphingomyelinase activity in the extract.