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.
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
2015-02-04; 0039-2499,Stroke - id: 1449962, year: 2015 Journal Article
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
2014-04-22; 1096-7192,Molecular genetics & metabolism - id: 884052, year: 2014 Journal Article
Lessons from everyday stroke care for clinical research and vice versa: comparison of a comprehensive and a research population of young stroke patients
Tanislav, Christian; Grittner, Ulrike; Misselwitz, Bjoern; Jungehuelsing, Gerhard Jan; Enzinger, Christian; von Sarnowski, Bettina; Putaala, Jukka; Kaps, Manfred; Kropp, Peter; Rolfs, Arndt; Tatlisumak, Turgut; Fazekas, Franz; Kolodny, Edwin; Norrving, Bo
2014-06-06; 1471-2377,BMC neurology - id: 1031822, year: 2014 Journal Article; Research Support, Non-U.S. Gov't
GM2 Gangliosidosis in British Jacob Sheep
Wessels, M E; Holmes, J P; Jeffrey, M; Jackson, M; Mackintosh, A; Kolodny, E H; Zeng, B J; Wang, C B; Scholes, S F E
2014-03-24; 0021-9975,Journal of comparative pathology - id: 851712, year: 2014 Journal Article
Headache as a symptom at stroke onset in 4,431 young ischaemic stroke patients. Results from the "stroke in young fabry patients (SIFAP1) study"
Kropp, Peter; Holzhausen, Martin; Kolodny, Edwin; Becker, Ulf; Dichgans, Martin; Diez-Tejedor, Exuperio; Enzinger, Christian; Fazekas, Franz; Fuentes, Blanca; Karpinska, Anna; Meyer, Wolfgang; Tanislav, Christian; Bottcher, Tobias; Rolfs, Arndt
2013-10-24; 0300-9564,Journal of neural transmission - id: 575582, year: 2013 Journal Article