Research Interests: Gene therapy for hemophilia, hemophilia associated joint disease (Hemophilia Arthropathy), by expressing bypassing agents (activated coagulation factor V, IX) via AAV vectors)

Research Interests: Dr. Young is a research scientist specializing in the field of Genetics and Metabolism. His main focus is molecular principles of auditory information processing and Gene Therapy approaches to treat neurological disorders.

Research Interests: Dr. Bobo’s research specializes in molecular mechanisms underlying rare congenital neurogenetic disorders and translating these insights into innovative gene therapies, with a primarily focus on lysosomal storage disorders (LSDs), including Mucopolysaccharidosis (MPS), and other severe neurodevelopmental conditions for which there are currently no effective treatments.

Research Interests: Dr. Hirsch works on Adeno-Associated Virus (AAV) biology and the development AAV Gene Therapeutics with a focus on Synthetic inverted terminal repeats, Ocular therapeutics, and DNA damage response, stemness, differentiation, and cancers.

Research Interests: The efficacy of the lentiviral vector system: Production systems, vector titer, vector specific activity, duration of transgene expression. The safety of the retro/lentiviral vector system: Vector mobilization, insertional mutagenesis, contamination with endogenous retroviruses. Basic HIV biology: The role of episomal HIV in the viral lifecycle, variations of RT products, HIV silencing, packaging signal mapping.

Research Interests: Intrinsic AAV proteins and their potential roles in AAV biology that can be applied to AAV gene therapies in the Central Nervous System (CNS).