Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.
Canavan disease, or Canavan–Van Bogaert–Bertrand disease, is a rare and fatal autosomal recessive degenerative disease that causes progressive damage to nerve cells and loss of white matter in the brain. It is one of the most common degenerative cerebral diseases of infancy. It is caused by a deficiency of the enzyme aminoacylase 2, and is one of a group of genetic diseases referred to as leukodystrophies. It is characterized by degeneration of myelin in the phospholipid layer insulating the axon of a neuron and is associated with a gene located on human chromosome 17.
Regenerative medicine deals with the "process of replacing, engineering or regenerating human or animal cells, tissues or organs to restore or establish normal function". This field holds the promise of engineering damaged tissues and organs by stimulating the body's own repair mechanisms to functionally heal previously irreparable tissues or organs.
The Miller School of Medicine, officially Leonard M. Miller School of Medicine, is the University of Miami's graduate medical school in Miami, Florida. Founded in 1952, it is the oldest medical school in the state of Florida.
The Nova Southeastern University College of Dental Medicine is the dental school of Nova Southeastern University. It is located in Fort Lauderdale, Florida, United States. When it opened in 1997, it was the first new dental school to open in the United States in 24 years. It is the largest dental school in Florida. The school is accredited by the American Dental Association.
Eva Lucille Feldman or Feldman is an American physician-scientist who is a leading authority on neurodegenerative disease. She serves as the Russell N. DeJong Professor of Neurology at the University of Michigan, as well as Director of the NeuroNetwork for Emerging Therapies and ALS Center of Excellence at Michigan Medicine. She was also named the James W. Albers Distinguished University Professor of Neurology.
The Llura Liggett Gund Award honors researchers for career achievements that have significantly advanced the research and development of preventions, treatments and cures for eye disease.
Ira Barrie Black was an American physician and neuroscientist who was an advocate of stem cell research and was the first director of the Stem Cell Institute of New Jersey at Robert Wood Johnson Medical School which was created to advance research in the field.
Sally Temple is an American developmental neuroscientist in Albany, New York. She is a co-founder and scientific director for The Neural Stem Cell Institute and is a professor of Neuroscience and Neuropharmacology at Albany Medical College Temple is also the principal investigator in her laboratory that focuses on neural stem cells and therapies for neurological-related disorders
HIV/AIDS research includes all medical research that attempts to prevent, treat, or cure HIV/AIDS, as well as fundamental research about the nature of HIV as an infectious agent and AIDS as the disease caused by HIV.
Alice M. Lazzarini is a scientist, author and researcher on neurogenetic disorders, including Huntington's disease and Parkinson's disease. She is an assistant professor of Neurology at Rutgers Robert Wood Johnson Medical School, where her work helped establish the genetic basis of Parkinson's. Later in life, she was diagnosed with Parkinson's—the very disease she had spent decades researching.
Jan A. Nolta is an American scientist and the director of the stem cell program at the UC Davis School of Medicine and Institute for Regenerative Cures. She is Scientific Director for the UC Davis Good Manufacturing Practice and editor of the journal Stem Cells. Nolta is known for her work with stem cell-related regenerative medicine. Nolta's current research focuses on treatment of Huntington's disease using mesenchymal stem cells. She was elected a AAAS Fellow in 2013.
Darwin Prockop was an American biochemist and progenitor cell researcher. He held academic posts at several universities, and joined the faculty at the Texas A&M Health Science Center in 2008. Prockop was elected to several academic societies, including the National Academy of Sciences and the Institute of Medicine.
Dr. John Engelhardt is the director at the University of Iowa Center for Gene Therapy of Cystic Fibrosis, as well as the head of the department of anatomy and cell biology. He is a well known scientist and inventor who created the first cloned ferret and has made huge strides in finding the cure for cystic fibrosis.
Melissa Helen Little is an Australian scientist and academic, currently Theme Director of Cell Biology, heading up the Kidney Regeneration laboratory at the Murdoch Children's Research Institute. She is also a Professor in the Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne, and Program Leader of Stem Cells Australia. In January 2022, she became CEO of the Novo Nordisk Foundation Center for Stem Cell Medicine reNEW, an international stem cell research center based at University of Copenhagen, and a collaboration between the University of Copenhagen, Denmark, Murdoch Children’s Research Institute, Australia, and Leiden University Medical Center, The Netherlands.
Graziella Pellegrini is an Italian Professor of Cell Biology and the Cell Therapy Program Coordinator at the University of Modena and Reggio Emilia. She has developed and championed cell therapy protocols in hospitals across Italy.
Kristin K. Baldwin is an American scientist who is a professor at the Department of Genetics and Development at Columbia University. Her research focuses on using reprogrammed and induced pluripotent stem cells to identify mechanisms and therapies related to human genetic risk for neurologic and cardiovascular disease. Her lab also studies how disease and aging affect the genome; they have used cloning to produce the first complete genome sequence of a single neuron and helped assess the effect of aging on induced pluripotent stem cells that may be used for cell therapies. They also design bespoke neuronal cells in a dish to understand brain function and disease. Baldwin's earlier work included being the first to clone a mouse from a neuron and being one of three groups to first produce an entire mouse from a skin cell by generating induced pluripotent stem cells. epigenetic changes of the genome and the brain.
Susan E. Quaggin is a Canadian nephrologist. She is the Charles Horace Mayo Professor of Medicine at Northwestern University Feinberg School of Medicine, Director of the Feinberg Cardiovascular Research Institute, prior chief of the Division of Nephrology, now Chair of the Department of Medicine.
Maria Grazia Roncarolo is an Italian pediatrician who is currently George D. Smith Professor in Stem Cell and Regenerative Medicine and Professor of Medicine at Stanford University. She is also the Director of the Stanford Institute of Stem Cell Biology and Regenerative Medicine along with Irving Weissman and Michael Longaker and the Director for Center for Definitive and Curative Medicine at Stanford.
Cynthia Dunbar is an American scientist and hematologist at the National Heart Lung and Blood Institute (NHLBI), which is part of the National Institutes of Health (NIH). She is the Branch Chief of the Translational Stem Cell Biology Branch.
