Cell and Gene Therapy

Center For Translational Neuromedicine
Division of Cell and Gene Therapy / Division of Glial Diseases and Therapeutics

Faculty

Major Clinical and Investigative Interests

The goal of our Center is to develop new approaches for treating neurological diseases, using cell and gene therapy. Our emphasis is on using these technologies to mobilize endogenous stem and progenitor cells of the adult brain and spinal cord, for purposes of structural repair. In addition, stem cell isolation and transplant strategies are also actively pursued. Our disease targets are those attributable to dysfunction or loss of single cell types, for instance dysmyelinating disease as a paradigm for the use of oligodendrocyte progenitor cells. The principal groups in the Center include Dr. Goldman, whose division focuses on neural and glial stem biology, and Dr. Nedergaard, whose division focuses on astrocytic physiology and pathology.

Goldman Lab

• Gene expression by progenitor cells of both the fetal and adult human CNS
• Glial progenitor stem cell-based therapy in pediatric leukodystophies and multiple sclerosis.
• Tumor stem cells and gliomas of the adult CNS
• Development of restricted neuronal progenitor cells for clinical transplantation and drug development, using human ES and PS cells.
• Inducing endogenous progenitors as a means of treatment in Huntington’s disease
• Defining the role of glial pathology in Huntington’s disease and psychopathologies
• Human glial chimeric mouse models of gliotropic viral infection

Nedergaard Lab

• The role of astrocytes in epileptogenesis and the treatment of seizure disorders
• The contribution of astrocytes to the regulation of cerebral blood flow, especially in ischemia
• Activity-dependent modulation of astrocytic signaling
• The role of gliosis and glial signaling in stroke
• Assessment of glial gene expression patterns in various disease states
• Developing new modalities for imaging native and transplanted glial progenitors in vivo
• Glial targeted treatment of spinal cord injury

Besides the senior faculty, the unit includes 10 junior faculty, and a significant representation of postdoctoral fellows and students. The labs are highly collaborative, with many investigators pursuing projects between the principal lab groups. The group’s work is currently supported by 6 NIH R01 grants and a P01 program project, as well as by a variety of foundations that include the National MS Society, the New York State Stem Cell Program, the New York State Spinal Cord Research Program, the Hereditary Disease Foundation, the CHDI Foundation, the Adelson Program in Neural Repair and Regeneration, the Mathers Charitable Foundation, and the James F. McDonnell Foundation. Past and present biotech and pharmaceutical collaborators include Sanofi-Aventis, Biogen, Regeneron, Merck, and others, advancing both the scope and translational intent of these studies.

Contact Person:	Steven Goldman, M.D., Ph.D. Maiken Nedergaard, M.D., DMSc.
Phone:	585-275-9550 (Dr. Goldman) 585-273-2868 (Dr. Nedergaard)
Fax:	585-276-2298
Email:	steven_goldman@urmc.rochester.edu nedergaard@urmc.rochester.edu