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Biology subjects

Schoenherr, C. J.

Publications and source records attributed to Schoenherr, C. J..

2 recordsLinked to original sources

Germline Targeted Baboon Apolipoprotein L-1 Protects Mice Against African Trypanosomes

Certain primates are immune to infection by most African trypanosome parasites due to apolipoprotein L-1 (APOL1), a primate-specific ion channel-forming protein. To broaden our understanding of primate APOL1, we generated a panel of trypanosome-resistant murine models expressing various primate APOL1 proteins. We used these mice to investigate the role of APOL1 in trypanosome immunity in vivo by challenging them with various human and livestock trypanosome isolates. Baboon APOL1 provides partial protection to trypanosome isolates, though its protective capacity was limited by poor expression. A more highly expressed chimeric APOL1 encoding human APOL1 with the baboon APOL1 C-terminus was protective against human-infective trypanosomes, although with a fitness cost likely associated with high expression. We investigated the long-standing assumption that human resistance to Trypanosoma vivax is mediated by APOL1. Surprisingly, APOL1-expressing mice were fully susceptible to T. vivax infection, challenging this hypothesis. These model systems are useful tools for evaluating the possibility of generating genetic engineered livestock for disease control.

microbiology↗

Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease mice

Lysosomal diseases are a class of genetic disorders predominantly caused by loss of lysosomal hydrolases, leading to lysosomal and cellular dysfunction. Enzyme Replacement Therapy (ERT), where recombinant enzyme is given intravenously, internalized by cells, and trafficked to the lysosome, has been applied to treat several lysosomal diseases. However, current ERT regimens do not correct disease phenotypes in all affected organs because the biodistribution of enzyme uptake does not match that of the affected cells and tissues that require the enzyme. We present here targeted ERT, an approach that utilizes antibody-enzyme fusion proteins to target the enzyme to specific tissues. The antibody moiety recognizes transmembrane proteins involved in lysosomal trafficking and that are also preferentially expressed in those cells most affected in disease. Using Pompe disease (PD) as an example, we show that targeted ERT is superior to ERT in treating the skeletal muscle phenotypes of PD mice.

bioengineering↗