Stem Cells and Gene Therapy

Characterization of the stem cell state and its control by comparative global gene expression and proteomics analyses.

Changes in specific genes that result in specific diseases, concentrating on Huntington disease and premature coronary artery disease.

Mammalian development, Transcriptional regulation and epigenetics, Hepatocyte differentiation, Heart valve formation, Signal transduction, Transgenic/knockout mice, Whole genome profiling

Genetic control and manipulation of hemopoiesis. Stem Cell Regulation. Retroviral Gene Transfer. Transgenic Mice.

Stem cells, developmental control, telomere biology, self-renewal and genetic instability.

Neurogenetics, Huntington disease and other triplet repeat disorders, transgenic/knockout mice, mouse models of human neurodegenerative disease, experimental therapeutics.

Role of imprinted genes in mammalian development. Epigenetics of embryonic stem cells and germ cell lineage. Gene targeting.

Genetic, genomic and comprehensive phenotyping studies for the autism spectrum disorders, idiopathic intellectual disabilities and other complex disorders of neurodevelopmental and/or behavioral disability.

Interplay between transcription, DNA methylation and histone modifications in embryonic stem cells and tumorigenesis.

Stem cells, Immunology, Inflammatory Disease, Cancer.

Pharmacogenomics of adverse drug reactions, genomics, personalized medicine.

Stem cells, regeneration, gene therapy, control of cell fate.

Gene-based therapies for diseases of the brain and eye, cell-type specific MiniPomoters for rAAV delivery of gene augmentation and genome editing (CRISPR/cas9) therapies to cure mouse models of the human disease.