Gene therapy is being developed in our laboratory as a tool in the treatment of malignant brain tumor, Parkinson's disease and the lysosomal storage diseases. Glioma cells transduced with recombinant retroviruses expressing cytosine deaminase become sensitive to the prodrug 5-fluorocytosine and this effect is enhanced by interferons. Using this same suicide gene approach in combination with a vector, expressing both cytosine deaminase and thymidine kinase has enabled us to significantly prolong the survival of rats with rapidly growing intracerebral glioma. We also participate in a consortium devoted to the use of another form of gene therapy, bone marrow transplantation, to successfully treat globoid cell leukodystrophy.
Our work in lysosomal storage diseases has also led us to discover molecular mimicry between antiglycolipid antibodies found in various autoimmune diseases such as multiple sclerosis and certain proteins including p24 and gp120 of the HIV virus and various brain cell membrane components. The gene for one such protein, GCP, has been cloned and its structural relationship to the galactolipids is being investigated.
Skeletal Muscle in Healthy Subjects versus Those with GNE-Related Myopathy: Evaluation with Shear-Wave US-A Pilot Study
Carpenter, Elizabeth L; Lau, Heather A; Kolodny, Edwin H; Adler, Ronald S. Skeletal Muscle in Healthy Subjects versus Those with GNE-Related Myopathy: Evaluation with Shear-Wave US-A Pilot Study. Radiology. 2015 Jun 2;277(2):546-554 (1816292)
Frequency of MELAS main mutation in a phenotype-targeted young ischemic stroke patient population
Tatlisumak, Turgut; Putaala, Jukka; Innila, Markus; Enzinger, Christian; Metso, Tiina M; Curtze, Sami; von Sarnowski, Bettina; Amaral-Silva, Alexandre; Jungehulsing, Gerhard Jan; Tanislav, Christian; Thijs, Vincent; Rolfs, Arndt; Norrving, Bo; Fazekas, Franz; Suomalainen, Anu; Kolodny, Edwin H. Frequency of MELAS main mutation in a phenotype-targeted young ischemic stroke patient population. Journal of neurology. 2015 Nov 14;263(2):257-262 (1834912)
Family History in Young Patients With Stroke
Thijs, Vincent; Grittner, Ulrike; Dichgans, Martin; Enzinger, Christian; Fazekas, Franz; Giese, Anne-Katrin; Kessler, Christof; Kolodny, Edwin; Kropp, Peter; Martus, Peter; Norrving, Bo; Ringelstein, Erich Bernd; Rothwell, Peter M; Schmidt, Reinhold; Tanislav, Christian; Tatlisumak, Turgut; von Sarnowski, Bettina; Rolfs, Arndt. Family History in Young Patients With Stroke. Stroke. 2015 Jun 2;46(7):1975-1978 (1640352)
Cerebrovascular involvement in fabry disease: current status of knowledge
Kolodny, Edwin; Fellgiebel, Andreas; Hilz, Max J; Sims, Katherine; Caruso, Paul; Phan, Thanh G; Politei, Juan; Manara, Renzo; Burlina, Alessandro. Cerebrovascular involvement in fabry disease: current status of knowledge. Stroke. 2014 Dec 9;46(1):302-313 (1449962)
GM1-gangliosidosis in American black bears: Clinical, pathological, biochemical and molecular genetic characterization
Muthupalani, Sureshkumar; Torres, Paola A; Wang, Betty C; Zeng, Bai Jin; Eaton, Samuel; Erdelyi, Ildiko; Ducore, Rebecca; Maganti, Rajanikarath; Keating, John; Perry, Bain J; Tseng, Florina S; Waliszewski, Nicole; Pokras, Mark; Causey, Robert; Seger, Rita; March, Philip; Tidwell, Amy; Pfannl, Rolf; Seyfried, Thomas; Kolodny, Edwin H; Alroy, Joseph. GM1-gangliosidosis in American black bears: Clinical, pathological, biochemical and molecular genetic characterization. Molecular genetics & metabolism. 2014 Feb 13;111(4):513-521 (884052)