Key Members
J.
Michael Mathis, Ph.D.
Director
Dept. of Cellular Biology and Anatomy
B. Jill Williams, Ph.D.
Associate Director
Dept. of Urology
Arrigo De Benedetti, Ph.D.
Dept. of Biochemistry
Ronald Klein, Ph.D.
Dept. of Pharmacology
Cherie-Ann Nathan, M.D.
Dept. of Otolaryngology
Kate Ryman, Ph.D.
Dept. of Microbiology and Immunology
Francesco Turturro, M.D.
Dept. of Medicine
Wei-Ming Duan, M.D., Ph.D.Dept.
of Cellular Biology and Anatomy
Benjamin Li, M.D.
Dept. of Surgery
Qian-Jin Zhang, Ph.D.
Dept. of Cellular Biology & Anatomy
Key Member
 |
Ronald Klein, Ph.D.
Dept. of Pharmacology
See the Curriculum
Vitae
Klein Lab (The Laboratory of Brain Gene Transfer)
My work involves
gene transfer to the brain for the purposes of gene therapy and other types
of functional studies, using the adeno-associated virus (AAV) vector system.
Because the recombinant vectors are replication-incompetent and results to
date in the rodent and non-human primate brain have not included any forms
of pathology, this system appears to be safe, and the transgene expression
is efficient and persistent.
Neurotrophic
factors are ideal therapeutic candidates to slow or reverse neurodegeneration,
but the peptide factors are difficult to apply to the brain in a targeted
and long-term manner. My lab studies gene delivery of NGF, BDNF, and GDNF
neurotrophic factors in animal lesioning models, monitoring therapeutic efficacy
at the level of neuroprotection, neurochemistry, and behavior.
Another
aim is to develop novel transgenic models of neurodegenerative diseases, such
as Alzheimer's and Parkinson's disease. While germ-line transgenic mice have
provided a wealth of information about the genetic bases of these diseases,
somatic cell gene transfer directly to brain sub-regions of adult animals
offers unique advantages for modeling. We have established models of neurofibrillary
tangles and Lewy pathology by expressing mutant forms of tau and alpha-synuclein,
respectively. These models can further our understanding of the disease process
as well be used to explore novel therapies, including gene therapy.
